C12Q 1/68 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions faisant intervenir des acides nucléiques
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (France)
Inventeur(s)
Cosette, Jérémie
Abrégé
The invention relates to a method for characterizing images of cell cultures in order to quantify cell parameters. The steps of the method comprise detecting at least one cell parameter or cell marker parameter in an image of cells in culture; constructing a mosaic of the image (squares or rectangles, also referred to as tiles); defining an image with smaller dimensions (also referred to as a density map) by integrating values of the cell parameters or cell marker parameters in each fraction of the mosaic; applying deep learning algorithms, which are implemented for example by a convolutional neural network, to the images generated in step c in order to determine a proximity score for the images of treated cells with respect to images of healthy cells or untreated diseased cells.
G06V 10/50 - Extraction de caractéristiques d’images ou de vidéos en effectuant des opérations dans des blocs d’imagesExtraction de caractéristiques d’images ou de vidéos en utilisant des histogrammes, p. ex. l’histogramme de gradient orienté [HoG]Extraction de caractéristiques d’images ou de vidéos en utilisant l’addition des valeurs d’intensité d’imageAnalyse de projection
G06V 10/82 - Dispositions pour la reconnaissance ou la compréhension d’images ou de vidéos utilisant la reconnaissance de formes ou l’apprentissage automatique utilisant les réseaux neuronaux
G06V 20/69 - Objets microscopiques, p. ex. cellules biologiques ou pièces cellulaires
05 - Produits pharmaceutiques, vétérinaires et hygièniques
09 - Appareils et instruments scientifiques et électriques
10 - Appareils et instruments médicaux
35 - Publicité; Affaires commerciales
36 - Services financiers, assurances et affaires immobilières
41 - Éducation, divertissements, activités sportives et culturelles
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Pharmaceutical, veterinary and sanitary products for medical
purposes; chemical preparations for medical and
pharmaceutical use or for veterinary use; biological
preparations for medical and pharmaceutical use or for
veterinary use; enzyme preparations for medical and
pharmaceutical use or for veterinary use; microorganism
cultures for medical and pharmaceutical use or for
veterinary use; bacteriological culture mediums for medical
or veterinary use; nutritional substances for
microorganisms; preparations of micro-organisms, of
genetically modified organisms and of cells for medical and
pharmaceutical use or for veterinary use, particularly for
gene therapy and cell therapy; compounds acting on genetic
or other pathologies, in human beings and animals,
particularly on muscle and neuromuscular diseases, diseases
of the immune system, cardiovascular diseases,
dermatological diseases, diseases of the central and
peripheral nervous system, cancers, asthma, allergies, lipid
disorders, obesity and diabetes and infectious diseases;
kits for preparing cells or for selecting biologically
active compounds; diagnostic products for medical or
veterinary use; diagnostic reagents for medical or
veterinary use; diagnostic reagents for in-vitro use in
biochemistry, clinical chemistry and microbiology; hygienic
products for medical use; diagnostic biomarking reagents for
medical use. Scientific and research apparatus and instruments; apparatus
and instruments for sound, image or data recording,
transmission, reproduction or processing; patient
(electronic) database; software; mobile applications;
downloadable Software as a Medical Device [SaMD];
downloadable computer programs; computer platforms in the
form of recorded or downloadable software. Surgical, medical apparatus and instruments; testing
apparatus for medical use; diagnostic apparatus for medical
use; cell culture instruments for medical use; surgical,
medical apparatus and instruments intended for use in gene
therapy and cell therapy; medical devices for neuromuscular
clinical research. Business management assistance; commercial business
appraisals; business management and organization
consultancy; business management consultancy; expertise in
commercial affairs; business information; business
inquiries; commercial administration of the licensing of
third-party goods and services; commercial or industrial
company management assistance; business management
assistance in scientific and technological project
development strategies; administrative management of
contracts relating to research; price comparison services;
compilation of statistics; collection of data in a central
file; systematization of data in a central file; computer
file management; market research and studies; organization
of fairs, exhibitions, trade fairs and job fairs for
commercial or advertising purposes; searching for industrial
or commercial partners; economic forecasting; sales
promotion for third parties; advertising; commercial
administration, commercial management advice, marketing
advice, commercial strategy advice, business management
advice, as well as office functions for the development and
innovation of companies in the health sector; business
feasibility studies, business appraisals, marketing study,
accounting, economic forecasting, and sponsorship search for
conducting development plans for companies in the health
sector; computerized file management for streamlining the
business branches of companies in the health sector;
administrative and commercial management and creation of
services enabling the organization of networks and matching
partners and/or service providers in areas of technological,
innovation expertise, financing various projects; database
management; compilation of information in computer
databases; consultancy and advisory services with respect to
company creation; advisory and assistance services in
company organization and management, particularly in the
context of a project selection, support and acceleration
program; sponsorship search; company audits (commercial
analyses). Providing financing for inventions; financing services in
the field of health; financial analysis; financial
appraisal; provision of financial information;
administration and management of grants for medical
research; providing financial subsidies; providing project
grants for health awareness projects; financial investments;
investment research; advice with respect to financing and
investment; fundraising services; raising or investing
capital; fund investment; searching for financial partners. Education; training; entertainment; sporting and cultural
activities; information relating to entertainment or
education; publication of books; organization of
competitions (education or entertainment); organization of
exhibitions for cultural or educational purposes; electronic
publication of books and journals online; electronic desktop
publishing; organizing and conducting training workshops,
colloquiums, conferences, congresses, seminars, symposiums
for cultural or educational purposes; planning of receptions
(entertainment); drafting and publication of texts other
than advertising texts; publishing the results of clinical
trials conducted on patients and scientific articles in
relation to clinical and genetic diseases; providing
non-downloadable electronic publications from a global
computer network or the Internet; print and electronic
magazine publishing services. Evaluations, assessments and research in the fields of
science and technology provided by engineers; appraisals and
assessments in the field of medical research; research in
the medical field; industrial analysis and research services
in the health sector; research and development of new
products (for others); technical project studies; scientific
research for medical purposes; clinical trials; medical
research laboratory services; biological, bacteriological,
gene and cell therapy, chemical and genetic research;
programming of software for Internet platforms; hosting of
platforms on the Internet; development of databases;
computer Platform as a Service [PaaS] comprising imaging and
physiology platforms [PaaS]; research and development of
pharmaceutical, veterinary and sanitary products for third
parties; sorting and selection of molecules; developing and
validating biological tests, cell tests, genetic tests and
immunological tests; fundamental and applied research in the
scientific, technological or industrial field; technical
research; activities in functional genomics for identifying,
characterizing, validating or developing biologically active
molecules; technical project study; professional consultancy
unrelated to business operations in the field of scientific
research and therapies. Medical and surgical services; medical and surgical
diagnosis; medical testing; hygienic and beauty care for
human beings; health services; advice with respect to
health; medical assistance; hospital services; nursing
homes; convalescence or rest homes; consultation with
respect to pharmacy; provision of information in the
pharmaceutical field; medical imaging services; human tissue
bank services; behavioral analysis for medical purposes;
provision of medical information; conducting medical
examinations; medical consultation services; medical testing
for the diagnosis and treatment of persons; genetic testing
services for medical purposes; telemedicine services;
therapeutic services.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
09 - Appareils et instruments scientifiques et électriques
10 - Appareils et instruments médicaux
35 - Publicité; Affaires commerciales
36 - Services financiers, assurances et affaires immobilières
41 - Éducation, divertissements, activités sportives et culturelles
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Pharmaceutical, veterinary and sanitary products for medical
purposes; chemical preparations for medical and
pharmaceutical use or for veterinary use; biological
preparations for medical and pharmaceutical use or for
veterinary use; enzyme preparations for medical and
pharmaceutical use or for veterinary use; microorganism
cultures for medical and pharmaceutical use or for
veterinary use; bacteriological culture mediums for medical
or veterinary use; nutritional substances for
microorganisms; preparations of micro-organisms, of
genetically modified organisms and of cells for medical and
pharmaceutical use or for veterinary use, particularly for
gene therapy and cell therapy; compounds acting on genetic
or other pathologies, in human beings and animals,
particularly on muscle and neuromuscular diseases, diseases
of the immune system, cardiovascular diseases,
dermatological diseases, diseases of the central and
peripheral nervous system, cancers, asthma, allergies, lipid
disorders, obesity and diabetes and infectious diseases;
kits for preparing cells or for selecting biologically
active compounds; diagnostic products for medical or
veterinary use; diagnostic reagents for medical or
veterinary use; diagnostic reagents for in-vitro use in
biochemistry, clinical chemistry and microbiology; hygienic
products for medical use; diagnostic biomarking reagents for
medical use. Scientific and research apparatus and instruments; apparatus
and instruments for sound, image or data recording,
transmission, reproduction or processing; patient
(electronic) database; software; mobile applications;
downloadable Software as a Medical Device [SaMD];
downloadable computer programs; computer platforms in the
form of recorded or downloadable software. Surgical, medical apparatus and instruments; testing
apparatus for medical use; diagnostic apparatus for medical
use; cell culture instruments for medical use; surgical,
medical apparatus and instruments intended for use in gene
therapy and cell therapy; medical devices for neuromuscular
clinical research. Business management assistance; commercial business
appraisals; business management and organization
consultancy; business management consultancy; expertise in
commercial affairs; business information; business
inquiries; commercial administration of the licensing of
third-party goods and services; commercial or industrial
company management assistance; business management
assistance in scientific and technological project
development strategies; administrative management of
contracts relating to research; price comparison services;
compilation of statistics; collection of data in a central
file; systematization of data in a central file; computer
file management; market research and studies; organization
of fairs, exhibitions, trade fairs and job fairs for
commercial or advertising purposes; searching for industrial
or commercial partners; economic forecasting; sales
promotion for third parties; advertising; commercial
administration, commercial management advice, marketing
advice, commercial strategy advice, business management
advice, as well as office functions for the development and
innovation of companies in the health sector; business
feasibility studies, business appraisals, marketing study,
accounting, economic forecasting, and sponsorship search for
conducting development plans for companies in the health
sector; computerized file management for streamlining the
business branches of companies in the health sector;
administrative and commercial management and creation of
services enabling the organization of networks and matching
partners and/or service providers in areas of technological,
innovation expertise, financing various projects; database
management; compilation of information in computer
databases; consultancy and advisory services with respect to
company creation; advisory and assistance services in
company organization and management, particularly in the
context of a project selection, support and acceleration
program; sponsorship search; company audits (commercial
analyses). Providing financing for inventions; financing services in
the field of health; financial analysis; financial
appraisal; provision of financial information;
administration and management of grants for medical
research; providing financial subsidies; providing project
grants for health awareness projects; financial investments;
investment research; advice with respect to financing and
investment; fundraising services; raising or investing
capital; fund investment; searching for financial partners. Education; training; entertainment; sporting and cultural
activities; information relating to entertainment or
education; publication of books; organization of
competitions (education or entertainment); organization of
exhibitions for cultural or educational purposes; electronic
publication of books and journals online; electronic desktop
publishing; organizing and conducting training workshops,
colloquiums, conferences, congresses, seminars, symposiums
for cultural or educational purposes; planning of receptions
(entertainment); drafting and publication of texts other
than advertising texts; publishing the results of clinical
trials conducted on patients and scientific articles in
relation to clinical and genetic diseases; providing
non-downloadable electronic publications from a global
computer network or the Internet; print and electronic
magazine publishing services. Evaluations, assessments and research in the fields of
science and technology provided by engineers; appraisals and
assessments in the field of medical research; research in
the medical field; industrial analysis and research services
in the health sector; research and development of new
products (for others); technical project studies; scientific
research for medical purposes; clinical trials; medical
research laboratory services; biological, bacteriological,
gene and cell therapy, chemical and genetic research;
programming of software for Internet platforms; hosting of
platforms on the Internet; development of databases;
computer Platform as a Service [PaaS] comprising imaging and
physiology platforms [PaaS]; research and development of
pharmaceutical, veterinary and sanitary products for third
parties; sorting and selection of molecules; developing and
validating biological tests, cell tests, genetic tests and
immunological tests; fundamental and applied research in the
scientific, technological or industrial field; technical
research; activities in functional genomics for identifying,
characterizing, validating or developing biologically active
molecules; technical project study; professional consultancy
unrelated to business operations in the field of scientific
research and therapies. Medical and surgical services; medical and surgical
diagnosis; medical testing; hygienic and beauty care for
human beings; health services; advice with respect to
health; medical assistance; hospital services; nursing
homes; convalescence or rest homes; consultation with
respect to pharmacy; provision of information in the
pharmaceutical field; medical imaging services; human tissue
bank services; behavioral analysis for medical purposes;
provision of medical information; conducting medical
examinations; medical consultation services; medical testing
for the diagnosis and treatment of persons; genetic testing
services for medical purposes; telemedicine services;
therapeutic services.
5.
CNS GENE DELIVERY USING PERIPHERAL ADMINISTRATION OF AAV VECTORS
CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (France)
Inventeur(s)
Barkats, Martine
Abrégé
The present invention relates to compositions and methods for the delivery of therapeutic proteins to the CNS using recombinant AAV vectors. More specifically, the invention relates to compositions and methods for delivering proteins into the cerebrospinal fluid of mammalian subjects through peripheral administration of AAV vectors. The invention may be used to treat various disorders of the central nervous system, including degenerative diseases and motor neuron diseases.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 25/00 - Médicaments pour le traitement des troubles du système nerveux
05 - Produits pharmaceutiques, vétérinaires et hygièniques
09 - Appareils et instruments scientifiques et électriques
10 - Appareils et instruments médicaux
35 - Publicité; Affaires commerciales
36 - Services financiers, assurances et affaires immobilières
41 - Éducation, divertissements, activités sportives et culturelles
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Pharmaceutical, veterinary and sanitary products for medical purposes; chemical preparations for medical and pharmaceutical use or for veterinary use; biological preparations for medical and pharmaceutical use or for veterinary use; enzyme preparations for medical and pharmaceutical use or for veterinary use; microorganism cultures for medical and pharmaceutical use or for veterinary use; bacteriological culture mediums for medical or veterinary use; nutritional substances for microorganisms; preparations of micro-organisms, of genetically modified organisms and of cells for medical and pharmaceutical use or for veterinary use, particularly for gene therapy and cell therapy; compounds acting on genetic or other pathologies, in human beings and animals, particularly on muscle and neuromuscular diseases, diseases of the immune system, cardiovascular diseases, dermatological diseases, diseases of the central and peripheral nervous system, cancers, asthma, allergies, lipid disorders, obesity and diabetes and infectious diseases; kits for preparing cells or for selecting biologically active compounds; diagnostic products for medical or veterinary use; diagnostic reagents for medical or veterinary use; diagnostic reagents for in-vitro use in biochemistry, clinical chemistry and microbiology; hygienic products for medical use; diagnostic biomarking reagents for medical use. Scientific and research apparatus and instruments; apparatus and instruments for sound, image or data recording, transmission, reproduction or processing; patient (electronic) database; software; mobile applications; downloadable Software as a Medical Device [SaMD]; downloadable computer programs; computer platforms in the form of recorded or downloadable software. Surgical, medical apparatus and instruments; testing apparatus for medical use; diagnostic apparatus for medical use; cell culture instruments for medical use; surgical, medical apparatus and instruments intended for use in gene therapy and cell therapy; medical devices for neuromuscular clinical research. Business management assistance; commercial business appraisals; business management and organization consultancy; business management consultancy; expertise in commercial affairs; business information; business inquiries; commercial administration of the licensing of third-party goods and services; commercial or industrial company management assistance; business management assistance in scientific and technological project development strategies; administrative management of contracts relating to research; price comparison services; compilation of statistics; collection of data in a central file; systematization of data in a central file; computer file management; market research and studies; organization of fairs, exhibitions, trade fairs and job fairs for commercial or advertising purposes; searching for industrial or commercial partners; economic forecasting; sales promotion for third parties; advertising; commercial administration, commercial management advice, marketing advice, commercial strategy advice, business management advice, as well as office functions for the development and innovation of companies in the health sector; business feasibility studies, business appraisals, marketing study, accounting, economic forecasting, and sponsorship search for conducting development plans for companies in the health sector; computerized file management for streamlining the business branches of companies in the health sector; administrative and commercial management and creation of services enabling the organization of networks and matching partners and/or service providers in areas of technological, innovation expertise, financing various projects; database management; compilation of information in computer databases; consultancy and advisory services with respect to company creation; advisory and assistance services in company organization and management, particularly in the context of a project selection, support and acceleration program; sponsorship search; company audits (commercial analyses). Providing financing for inventions; financing services in the field of health; financial analysis; financial appraisal; provision of financial information; administration and management of grants for medical research; providing financial subsidies; providing project grants for health awareness projects; financial investments; investment research; advice with respect to financing and investment; fundraising services; raising or investing capital; fund investment; searching for financial partners. Education; training; entertainment; sporting and cultural activities; information relating to entertainment or education; publication of books; organization of competitions (education or entertainment); organization of exhibitions for cultural or educational purposes; electronic publication of books and journals online; electronic desktop publishing; organizing and conducting training workshops, colloquiums, conferences, congresses, seminars, symposiums for cultural or educational purposes; planning of receptions (entertainment); drafting and publication of texts other than advertising texts; publishing the results of clinical trials conducted on patients and scientific articles in relation to clinical and genetic diseases; providing non-downloadable electronic publications from a global computer network or the Internet; print and electronic magazine publishing services. Evaluations, assessments and research in the fields of science and technology provided by engineers; appraisals and assessments in the field of medical research; research in the medical field; industrial analysis and research services in the health sector; research and development of new products (for others); technical project studies; scientific research for medical purposes; clinical trials; medical research laboratory services; biological, bacteriological, gene and cell therapy, chemical and genetic research; programming of software for Internet platforms; hosting of platforms on the Internet; development of databases; computer Platform as a Service [PaaS] comprising imaging and physiology platforms [PaaS]; research and development of pharmaceutical, veterinary and sanitary products for third parties; sorting and selection of molecules; developing and validating biological tests, cell tests, genetic tests and immunological tests; fundamental and applied research in the scientific, technological or industrial field; technical research; activities in functional genomics for identifying, characterizing, validating or developing biologically active molecules; technical project study; professional consultancy unrelated to business operations in the field of scientific research and therapies. Medical and surgical services; medical and surgical diagnosis; medical testing; hygienic and beauty care for human beings; health services; advice with respect to health; medical assistance; hospital services; nursing homes; convalescence or rest homes; consultation with respect to pharmacy; provision of information in the pharmaceutical field; medical imaging services; human tissue bank services; behavioral analysis for medical purposes; provision of medical information; conducting medical examinations; medical consultation services; medical testing for the diagnosis and treatment of persons; genetic testing services for medical purposes; telemedicine services; therapeutic services.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
09 - Appareils et instruments scientifiques et électriques
10 - Appareils et instruments médicaux
35 - Publicité; Affaires commerciales
36 - Services financiers, assurances et affaires immobilières
41 - Éducation, divertissements, activités sportives et culturelles
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Pharmaceutical, veterinary and sanitary products for medical purposes; chemical preparations for medical and pharmaceutical use or for veterinary use; biological preparations for medical and pharmaceutical use or for veterinary use; enzyme preparations for medical and pharmaceutical use or for veterinary use; microorganism cultures for medical and pharmaceutical use or for veterinary use; bacteriological culture mediums for medical or veterinary use; nutritional substances for microorganisms; preparations of micro-organisms, of genetically modified organisms and of cells for medical and pharmaceutical use or for veterinary use, particularly for gene therapy and cell therapy; compounds acting on genetic or other pathologies, in human beings and animals, particularly on muscle and neuromuscular diseases, diseases of the immune system, cardiovascular diseases, dermatological diseases, diseases of the central and peripheral nervous system, cancers, asthma, allergies, lipid disorders, obesity and diabetes and infectious diseases; kits for preparing cells or for selecting biologically active compounds; diagnostic products for medical or veterinary use; diagnostic reagents for medical or veterinary use; diagnostic reagents for in-vitro use in biochemistry, clinical chemistry and microbiology; hygienic products for medical use; diagnostic biomarking reagents for medical use. Scientific and research apparatus and instruments; apparatus and instruments for sound, image or data recording, transmission, reproduction or processing; patient (electronic) database; software; mobile applications; downloadable Software as a Medical Device [SaMD]; downloadable computer programs; computer platforms in the form of recorded or downloadable software. Surgical, medical apparatus and instruments; testing apparatus for medical use; diagnostic apparatus for medical use; cell culture instruments for medical use; surgical, medical apparatus and instruments intended for use in gene therapy and cell therapy; medical devices for neuromuscular clinical research. Business management assistance; commercial business appraisals; business management and organization consultancy; business management consultancy; expertise in commercial affairs; business information; business inquiries; commercial administration of the licensing of third-party goods and services; commercial or industrial company management assistance; business management assistance in scientific and technological project development strategies; administrative management of contracts relating to research; price comparison services; compilation of statistics; collection of data in a central file; systematization of data in a central file; computer file management; market research and studies; organization of fairs, exhibitions, trade fairs and job fairs for commercial or advertising purposes; searching for industrial or commercial partners; economic forecasting; sales promotion for third parties; advertising; commercial administration, commercial management advice, marketing advice, commercial strategy advice, business management advice, as well as office functions for the development and innovation of companies in the health sector; business feasibility studies, business appraisals, marketing study, accounting, economic forecasting, and sponsorship search for conducting development plans for companies in the health sector; computerized file management for streamlining the business branches of companies in the health sector; administrative and commercial management and creation of services enabling the organization of networks and matching partners and/or service providers in areas of technological, innovation expertise, financing various projects; database management; compilation of information in computer databases; consultancy and advisory services with respect to company creation; advisory and assistance services in company organization and management, particularly in the context of a project selection, support and acceleration program; sponsorship search; company audits (commercial analyses). Providing financing for inventions; financing services in the field of health; financial analysis; financial appraisal; provision of financial information; administration and management of grants for medical research; providing financial subsidies; providing project grants for health awareness projects; financial investments; investment research; advice with respect to financing and investment; fundraising services; raising or investing capital; fund investment; searching for financial partners. Education; training; entertainment; sporting and cultural activities; information relating to entertainment or education; publication of books; organization of competitions (education or entertainment); organization of exhibitions for cultural or educational purposes; electronic publication of books and journals online; electronic desktop publishing; organizing and conducting training workshops, colloquiums, conferences, congresses, seminars, symposiums for cultural or educational purposes; planning of receptions (entertainment); drafting and publication of texts other than advertising texts; publishing the results of clinical trials conducted on patients and scientific articles in relation to clinical and genetic diseases; providing non-downloadable electronic publications from a global computer network or the Internet; print and electronic magazine publishing services. Evaluations, assessments and research in the fields of science and technology provided by engineers; appraisals and assessments in the field of medical research; research in the medical field; industrial analysis and research services in the health sector; research and development of new products (for others); technical project studies; scientific research for medical purposes; clinical trials; medical research laboratory services; biological, bacteriological, gene and cell therapy, chemical and genetic research; programming of software for Internet platforms; hosting of platforms on the Internet; development of databases; computer Platform as a Service [PaaS] comprising imaging and physiology platforms [PaaS]; research and development of pharmaceutical, veterinary and sanitary products for third parties; sorting and selection of molecules; developing and validating biological tests, cell tests, genetic tests and immunological tests; fundamental and applied research in the scientific, technological or industrial field; technical research; activities in functional genomics for identifying, characterizing, validating or developing biologically active molecules; technical project study; professional consultancy unrelated to business operations in the field of scientific research and therapies. Medical and surgical services; medical and surgical diagnosis; medical testing; hygienic and beauty care for human beings; health services; advice with respect to health; medical assistance; hospital services; nursing homes; convalescence or rest homes; consultation with respect to pharmacy; provision of information in the pharmaceutical field; medical imaging services; human tissue bank services; behavioral analysis for medical purposes; provision of medical information; conducting medical examinations; medical consultation services; medical testing for the diagnosis and treatment of persons; genetic testing services for medical purposes; telemedicine services; therapeutic services.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
09 - Appareils et instruments scientifiques et électriques
10 - Appareils et instruments médicaux
35 - Publicité; Affaires commerciales
36 - Services financiers, assurances et affaires immobilières
41 - Éducation, divertissements, activités sportives et culturelles
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
(1) Produits pharmaceutiques, vétérinaires et hygiéniques pour la médecine; préparations chimiques à usage médical, pharmaceutique ou à usage vétérinaire; préparations biologiques à usage médical, pharmaceutique ou à usage vétérinaire; préparations enzymatiques à usage médical, pharmaceutique ou à usage vétérinaire; cultures de micro-organismes à usage médical, pharmaceutique ou vétérinaire; bouillons de culture pour la bactériologie à usage médical ou vétérinaire; substances nutritives pour micro-organismes; préparations de micro-organismes, d'organismes génétiquement modifiées et de cellules à usage médical, pharmaceutique ou à usage vétérinaire, en particulier pour la thérapie génique et la thérapie cellulaire; composés actifs sur les pathologies génétiques ou non, des êtres humains et des animaux, notamment sur les maladies musculaires, neuromusculaires, du système immunitaire, cardio-vasculaires, les maladies dermatologiques, les maladies du système nerveux central et périphérique, les cancers, l'asthme, les allergies, les désordres lipidiques, l'obésité et le diabète, les maladies infectieuses; kits pour la préparation de cellules ou pour la sélection de composés biologiquement actifs; produits pour le diagnostic à usage médical ou vétérinaire; réactifs pour le diagnostic à usage médical ou vétérinaire; réactifs pour le diagnostic pour un usage in vitro en biochimie, chimie clinique et en microbiologie; produits hygiéniques à usage médical; Réactifs de biomarquage pour le diagnostic à usage médical.
(2) Appareils et instruments scientifiques, de recherche; appareils et instruments pour l'enregistrement, la transmission, la reproduction ou le traitement de sons, d'images ou de données; base de données (électronique) de patients; logiciels; applications mobiles; logiciels en tant que dispositifs médicaux [SaMD] téléchargeables; programmes informatiques téléchargeables; plateformes informatiques sous forme de logiciels enregistrés ou téléchargeables.
(3) Appareils et instruments chirurgicaux, médicaux; appareils pour l'analyse à usage médical; appareils pour le diagnostic à usage médical; instruments de culture cellulaire à usage médical; appareils et instruments chirurgicaux, médicaux destinés à la thérapie génique et la thérapie cellulaire; dispositifs médicaux pour la recherche clinique neuromusculaire. (1) Aide à la direction des affaires; estimations en affaires commerciales; conseils en organisation et direction des affaires; consultations pour la direction des affaires; expertises en affaires commerciales; informations d'affaires; renseignements d'affaires; administration commerciale de licences de produits et de services de tiers; aide à la direction d'entreprises industrielles ou commerciales; aide à la direction des affaires en stratégies de développement de projets scientifiques et technologiques; gestion administrative de contrats en lien avec la recherche; services de comparaison de prix; établissement de statistiques; recueil de données dans un fichier central; systématisation de données dans un fichier central; gestion de fichiers informatiques; études et recherches de marchés; organisation de salons, d'expositions, de foires à buts commerciaux ou de publicité; recherche de partenaires industriels ou commerciaux; prévisions économiques; promotion des ventes pour des tiers; publicité; administration commerciale, prestations de conseils en gestion commerciale, prestations de conseils en marketing, prestations de conseil en stratégie commerciale, prestations de conseils en direction des affaires, ainsi que travaux de bureau pour le développement et l'innovation des entreprises dans le secteur de la santé; étude de faisabilité commerciale, estimation d'affaire commerciale, étude de marché, comptabilité, prévisions économiques, et recherche de parraineurs pour la réalisation de plans de développement à destination des entreprises du secteur de la santé; gestion de fichiers informatiques pour la rationalisation des branches d'activité des entreprises du secteur de la santé; services de gestion et de création administrative et commerciale de prestations permettant l'organisation de réseaux et de mise en contact de partenaires et/ou prestataires sur des pôles de compétences technologiques, d'innovation, de financement de projets divers; gestion de bases de données; services de compilation de données dans des bases de données informatiques; services de consultations et de conseils en matière de création d'entreprise; services de conseils et d'aide à l'organisation et la direction aux entreprises, notamment dans le cadre d'un programme de sélection, d'accompagnement et d'accélération de projets; recherche de parraineurs; audits d'entreprises (analyses commerciales).
(2) Mise à disposition de financement pour des inventions; services de financement dans le domaine de la santé; analyse financière; estimation financière; mise à disposition d'informations financières; administration et gestion de subventions pour la recherche médicale; mise à disposition de subventions financières; mise à disposition de subventions de projets dans le cadre de projets de sensibilisation aux questions de santé; investissements financiers; recherche en investissement; conseils en matière de financement et d'investissement; collectes de fonds; constitution ou investissement de capitaux; placement de fonds; recherche de partenaires financiers.
(3) Éducation; formation; divertissement; activités sportives et culturelles; informations en matière de divertissement ou d'éducation; publication de livres; organisation de concours (éducation ou divertissement); organisation d'expositions à buts culturels ou éducatifs; publication électronique de livres et de périodiques en ligne; micro-édition; organisation et conduite d'ateliers de formation, de colloques, de conférences, de congrès, de séminaires, de symposiums à des fins culturelles ou éducatives; planification de réceptions (divertissement); rédaction et publication de textes autres que textes publicitaires; publication de résultats d'essais cliniques réalisés sur des malades et d'articles scientifiques en rapport avec les maladies cliniques et génétiques; mise à disposition de publications électroniques, non téléchargeables, à partir d'un réseau informatique mondial ou d'Internet; services de publication de magazines papier et sous forme électronique.
(4) Evaluations, estimations et recherches dans les domaines scientifique et technologique rendues par des ingénieurs; évaluations et estimations dans le domaine de la recherche médicale; recherches dans le domaine médical; services d'analyse et de recherches industrielles dans le secteur de la santé; recherche et développement de nouveaux produits (pour des tiers); études de projets techniques; recherches scientifiques à but médical; essais cliniques; services de laboratoire de recherche médicale; recherches biologiques, en bactériologie, en thérapie génique et cellulaire, en chimie et en génétique; programmation de logiciels pour des plateformes Internet; hébergement de plateformes sur Internet; développement de bases de données; plateforme informatique en tant que service [PaaS] comprenant des plateformes d'imagerie et de physiologie (PaaS); services de recherche et développement de produits pharmaceutiques, vétérinaires et hygiéniques pour des tiers; triage et sélection de molécules; développement et validation de tests biologiques, de tests cellulaires, de tests génétiques et de tests immunologiques; recherche fondamentale et appliquée dans le domaine scientifique, technologique ou industrielle; recherches techniques; activités de génomique fonctionnelle, pour identifier, caractériser, valider ou développer des molécules biologiquement actives; étude de projets techniques; consultations professionnelles sans rapport avec la conduite des affaires dans le domaine de la recherche scientifique et des thérapies.
(5) Services médicaux et chirurgicaux; diagnostic médical et chirurgical; tests médicaux; soins d'hygiène et de beauté pour êtres humains; services de santé; conseils en matière de santé; assistance médicale; services hospitaliers; maisons médicalisées; maisons de convalescence ou de repos; consultation en matière de pharmacie; mise à disposition d'informations dans le domaine pharmaceutique; services d'imagerie médicale; services de banque de tissus humains; analyses comportementales à des fins médicales; mise à disposition d'informations médicales; réalisation d'examens médicaux; services de consultations médicales; analyses médicales pour le diagnostic et le traitement de personnes; services de dépistage génétique à des fins médicales; services de télémédecine; services thérapeutiques.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
09 - Appareils et instruments scientifiques et électriques
10 - Appareils et instruments médicaux
35 - Publicité; Affaires commerciales
36 - Services financiers, assurances et affaires immobilières
41 - Éducation, divertissements, activités sportives et culturelles
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
(1) Produits pharmaceutiques, vétérinaires et hygiéniques pour la médecine; préparations chimiques à usage médical, pharmaceutique ou à usage vétérinaire; préparations biologiques à usage médical, pharmaceutique ou à usage vétérinaire; préparations enzymatiques à usage médical, pharmaceutique ou à usage vétérinaire; cultures de micro-organismes à usage médical, pharmaceutique ou vétérinaire; bouillons de culture pour la bactériologie à usage médical ou vétérinaire; substances nutritives pour micro-organismes; préparations de micro-organismes, d'organismes génétiquement modifiées et de cellules à usage médical, pharmaceutique ou à usage vétérinaire, en particulier pour la thérapie génique et la thérapie cellulaire; composés actifs sur les pathologies génétiques ou non, des êtres humains et des animaux, notamment sur les maladies musculaires, neuromusculaires, du système immunitaire, cardio-vasculaires, les maladies dermatologiques, les maladies du système nerveux central et périphérique, les cancers, l'asthme, les allergies, les désordres lipidiques, l'obésité et le diabète, les maladies infectieuses; kits pour la préparation de cellules ou pour la sélection de composés biologiquement actifs; produits pour le diagnostic à usage médical ou vétérinaire; réactifs pour le diagnostic à usage médical ou vétérinaire; réactifs pour le diagnostic pour un usage in vitro en biochimie, chimie clinique et en microbiologie; produits hygiéniques à usage médical; réactifs de biomarquage pour le diagnostic à usage médical.
(2) Appareils et instruments scientifiques, de recherche; appareils et instruments pour l'enregistrement, la transmission, la reproduction ou le traitement de sons, d'images ou de données; base de données (électronique) de patients; logiciels; applications mobiles; logiciels en tant que dispositifs médicaux [SaMD] téléchargeables; programmes informatiques téléchargeables; plateformes informatiques sous forme de logiciels enregistrés ou téléchargeables.
(3) Appareils et instruments chirurgicaux, médicaux; appareils pour l'analyse à usage médical; appareils pour le diagnostic à usage médical; instruments de culture cellulaire à usage médical; appareils et instruments chirurgicaux, médicaux destinés à la thérapie génique et la thérapie cellulaire; dispositifs médicaux pour la recherche clinique neuromusculaire. (1) Aide à la direction des affaires; estimations en affaires commerciales; conseils en organisation et direction des affaires; consultations pour la direction des affaires; expertises en affaires commerciales; informations d'affaires; renseignements d'affaires; administration commerciale de licences de produits et de services de tiers; aide à la direction d'entreprises industrielles ou commerciales; aide à la direction des affaires en stratégies de développement de projets scientifiques et technologiques; gestion administrative de contrats en lien avec la recherche; services de comparaison de prix; établissement de statistiques; recueil de données dans un fichier central; systématisation de données dans un fichier central; gestion de fichiers informatiques; études et recherches de marchés; organisation de salons, d'expositions, de foires à buts commerciaux ou de publicité; recherche de partenaires industriels ou commerciaux; prévisions économiques; promotion des ventes pour des tiers; publicité; administration commerciale, prestations de conseils en gestion commerciale, prestations de conseils en marketing, prestations de conseil en stratégie commerciale, prestations de conseils en direction des affaires, ainsi que travaux de bureau pour le développement et l'innovation des entreprises dans le secteur de la santé; étude de faisabilité commerciale, estimation d'affaire commerciale, étude de marché, comptabilité, prévisions économiques, et recherche de parraineurs pour la réalisation de plans de développement à destination des entreprises du secteur de la santé; gestion de fichiers informatiques pour la rationalisation des branches d'activité des entreprises du secteur de la santé; services de gestion et de création administrative et commerciale de prestations permettant l'organisation de réseaux et de mise en contact de partenaires et/ou prestataires sur des pôles de compétences technologiques, d'innovation, de financement de projets divers; gestion de bases de données; services de compilation de données dans des bases de données informatiques; services de consultations et de conseils en matière de création d'entreprise; services de conseils et d'aide à l'organisation et la direction aux entreprises, notamment dans le cadre d'un programme de sélection, d'accompagnement et d'accélération de projets; recherche de parraineurs; audits d'entreprises (analyses commerciales).
(2) Mise à disposition de financement pour des inventions; services de financement dans le domaine de la santé; analyse financière; estimation financière; mise à disposition d'informations financières; administration et gestion de subventions pour la recherche médicale; mise à disposition de subventions financières; mise à disposition de subventions de projets dans le cadre de projets de sensibilisation aux questions de santé; investissements financiers; recherche en investissement; conseils en matière de financement et d'investissement; collectes de fonds; constitution ou investissement de capitaux; placement de fonds; recherche de partenaires financiers.
(3) Éducation; formation; divertissement; activités sportives et culturelles; informations en matière de divertissement ou d'éducation; publication de livres; organisation de concours (éducation ou divertissement); organisation d'expositions à buts culturels ou éducatifs; publication électronique de livres et de périodiques en ligne; micro-édition; organisation et conduite d'ateliers de formation, de colloques, de conférences, de congrès, de séminaires, de symposiums à des fins culturelles ou éducatives; planification de réceptions (divertissement); rédaction et publication de textes autres que textes publicitaires; publication de résultats d'essais cliniques réalisés sur des malades et d'articles scientifiques en rapport avec les maladies cliniques et génétiques; mise à disposition de publications électroniques, non téléchargeables, à partir d'un réseau informatique mondial ou d'Internet; services de publication de magazines papier et sous forme électronique.
(4) Evaluations, estimations et recherches dans les domaines scientifique et technologique rendues par des ingénieurs; évaluations et estimations dans le domaine de la recherche médicale; recherches dans le domaine médical; services d'analyse et de recherches industrielles dans le secteur de la santé; recherche et développement de nouveaux produits (pour des tiers); études de projets techniques; recherches scientifiques à but médical; essais cliniques; services de laboratoire de recherche médicale; recherches biologiques, en bactériologie, en thérapie génique et cellulaire, en chimie et en génétique; programmation de logiciels pour des plateformes Internet; hébergement de plateformes sur Internet; développement de bases de données; plateforme informatique en tant que service [PaaS] comprenant des plateformes d'imagerie et de physiologie (PaaS); services de recherche et développement de produits pharmaceutiques, vétérinaires et hygiéniques pour des tiers; triage et sélection de molécules; développement et validation de tests biologiques, de tests cellulaires, de tests génétiques et de tests immunologiques; recherche fondamentale et appliquée dans le domaine scientifique, technologique ou industrielle; recherches techniques; activités de génomique fonctionnelle, pour identifier, caractériser, valider ou développer des molécules biologiquement actives; étude de projets techniques; consultations professionnelles sans rapport avec la conduite des affaires dans le domaine de la recherche scientifique et des thérapies.
(5) Services médicaux et chirurgicaux; diagnostic médical et chirurgical; tests médicaux; soins d'hygiène et de beauté pour êtres humains; services de santé; conseils en matière de santé; assistance médicale; services hospitaliers; maisons médicalisées; maisons de convalescence ou de repos; consultation en matière de pharmacie; mise à disposition d'informations dans le domaine pharmaceutique; services d'imagerie médicale; services de banque de tissus humains; analyses comportementales à des fins médicales; mise à disposition d'informations médicales; réalisation d'examens médicaux; services de consultations médicales; analyses médicales pour le diagnostic et le traitement de personnes; services de dépistage génétique à des fins médicales; services de télémédecine; services thérapeutiques.
11.
USE OF BAZEDOXIFENE FOR INCREASING MUSCLE SURVIVAL
INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) (France)
Inventeur(s)
Nissan, Xavier
Bruge, Céline
Pellier, Emilie
Richard, Isabelle
Abrégé
The invention concerns the use of bazedoxifene as a representative of third generation SERMs, namely compounds comprising a 2-phenylindole group, in order to increase muscle survival, in particular in the treatment of dysferlinopathies.
A61K 31/55 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p. ex. guanéthidine ou rifamycines ayant des cycles à sept chaînons, p. ex. azélastine, pentylènetétrazole
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (France)
Inventeur(s)
Buj-Bello, Ana
Renaud-Gabardos, Edith
Veron, Philippe
Abrégé
The invention relates to a combination of a recombinant AAVpo1 vector and a recombinant AAV9, AAVrh10 or AAVrh74 vector for use in the gene therapy of muscle and/or nervous system disorders in an individual in need thereof.
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE (France)
SORBONNE UNIVERSITÉ (France)
UNIVERSITE D'EVRY VAL D'ESSONNE (France)
ASSOCIATION INSTITUT DE MYOLOGIE (France)
Inventeur(s)
Mingozzi, Federico
Colella, Pasqualina
Abrégé
The present invention relates to hybrid transcription regulatory elements to drive gene expression, in particular hybrid promoters, designed by the fusion of at least two transcription regulatory elements with different tissue selectivity, such as two promoters driving expression in different tissues in a tissue-selective manner.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
Institut National de la Sante et de la Recherche Medicale (France)
Universite d'Evry Val d'Essonne (France)
Inventeur(s)
Ronzitti, Giuseppe
Jauze, Louisa
Charles, Séverine
Mingozzi, Federico
Abrégé
The invention relates to a nucleic acid construct for gene therapy of FGF-23 related hypophosphatemic diseases, in particular gene therapy directed to muscle, liver or hematopoietic tissue, more particularly liver tissue. The invention relates also to a vector comprising the nucleic acid construct, and their use for the treatment of FGF-23 related hypophosphatemic diseases, in particular XLH, by gene therapy.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61K 38/18 - Facteurs de croissanceRégulateurs de croissance
A61P 7/00 - Médicaments pour le traitement des troubles du sang ou du fluide extracellulaire
INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) (France)
UNIVERSITE D'EVRY-VAL-D'ESSONNE (France)
Inventeur(s)
Guiraud, Simon
Abrégé
The present invention concerns a micro-utrophin comprising the structure NTDH1R1R2R3H2R22H4CRD and two further rod domains selected from the group consisting of: - the R4 to R10 rod domains of utrophin; - the R16 and R17 rod domains of dystrophin; a nucleic acid sequence encoding such a micro-utrophin, a recombinant adeno-associated virus (rAAV) vector comprising such a sequence, and their use for treating a muscular dystrophy such as Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD).
A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) (France)
UNIVERSITE D'EVRY-VAL-D'ESSONNE (France)
Inventeur(s)
Albini, Sonia
Palmieri, Laura
Richard, Isabelle
Abrégé
The present invention concerns mididystrophins comprising at least the rod domains R1, R8, R9, R10, R11, R12, R16, R17 and R24 of the dystrophin and their use for treating a muscular dystrophy, in particular a Duchenne muscular dystrophy (DMD).
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
C12N 5/10 - Cellules modifiées par l'introduction de matériel génétique étranger, p. ex. cellules transformées par des virus
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 15/62 - Séquences d'ADN codant pour des protéines de fusion
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
18.
N-TERMINAL TRUNCATED GLYCOGEN DEBRANCHING ENZYMES FOR THE TREATMENT OF GLYCOGEN STORAGE DISEASE III
A61K 38/00 - Préparations médicinales contenant des peptides
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 9/44 - Hydrolases (3.) agissant sur les composés glycosyliques (3.2) agissant sur les liaisons alpha-glucosidiques-1, 6, p. ex. iso-amylase, pullulanase
C12N 15/52 - Gènes codant pour des enzymes ou des proenzymes
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS) (France)
GENETHON (France)
Inventeur(s)
Gazeau, Florence
Silva, Amanda Karine Andriola
Merten, Otto-Wilhelm
Wilhelm, Claire
Piffoux, Max
Abrégé
A fluidic system for producing extracellular vesicles from producer cells, including at least one container, a liquid medium contained by the container and producer cells, characterised in that it also includes microcarriers suspended in the liquid medium, the majority of producer cells being adherent to the surface of the microcarriers, and a liquid medium agitator, the agitator and the dimensions of the container being adapted to control a turbulent flow of the liquid medium in the container.
C12M 3/06 - Appareillage pour la culture de tissus, de cellules humaines, animales ou végétales, ou de virus avec des moyens de filtration, d'ultrafiltration, d'osmose inverse ou de dialyse
C12M 1/00 - Appareillage pour l'enzymologie ou la microbiologie
C12M 1/12 - Appareillage pour l'enzymologie ou la microbiologie avec des moyens de stérilisation, filtration ou dialyse
CENTRE D'ETUDE DES CELLULES SOUCHES (CECS) (France)
GENETHON (France)
Inventeur(s)
Martin, Samia
Boissart, Claire
Benchoua, Alexandra
Abrégé
The invention relates to a lentiviral vector comprising a tricistronic expression cassette encoding: - tyrosine hydroxylase of SEQ ID NO 1; - GTP cyclohydrolase I of SEQ ID NO 2; and - aromatic L-amino acid decarboxylase of SEQ ID NO 3, or a functional variant of said enzymes having a nucleic sequence that is at least 80% identical to SEQ ID NOs 1, 2 and 3. The vector is characterised in that the three enzymes are expressed under the control of the same human PGK-1 promoter, or a functional variant of said human PGK-1 promoter having a nucleic sequence that is at least 80% identical to SEQ ID NO 4.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
The present invention relates to the generation and use of machine learning models in order to predict some characteristics of a phenotype of a biological object such as an adeno-associated virus. It also relates to the preparation of a reduced training dataset for improving the efficiency and the explicability of the machine learning models generated.
INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) (France)
Inventeur(s)
Ronzitti, Giuseppe
Vidal, Pauline
Abrégé
The present invention relates to novel hybrid promoters. The invention further relates to expression cassettes and vectors containing said hybrid promoters. Also disclosed herein are methods implementing these hybrid promoters, in particular methods of gene therapy.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
INSERM (Institut National de la Santé et de la Recherche Médicale) (France)
Universite d'Evry Val d'Essonne (France)
Sorbonne Universite (France)
Association Institut de Myologie (France)
Inventeur(s)
Richard, Isabelle
Gicquel, Evelyne
Mingozzi, Federico
Abrégé
The invention relates to a recombinant adeno-associated virus (AAV) capsid protein, which is a hybrid between AAV serotype 9 (AAV9) and AAV serotype 74 (AAVrh74) capsid proteins, wherein said recombinant hybrid AAV capsid protein has a reduced liver tropism compared to the parent AAV9 and AAVrh74 capsid proteins. The invention relates also to the derived hybrid AAV serotype vector particles packaging a gene of interest and their use in gene therapy, in particular for treating neuromuscular genetic diseases.
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE (France)
Inventeur(s)
Ronzitti, Giuseppe
Vidal, Pauline
Abrégé
The invention relates to an AAV vector comprising the combination of a tandem liver-muscle selective promoter and a liver detargeted AAV capsid protein modified with a muscle-targeting peptide and its use in gene therapy of muscle diseases, in particular genetic neuromuscular diseases.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
Institut National de la Santé et de la Recherche Médicale (France)
Universite d'Evry Val d'Essonne (France)
Inventeur(s)
Richard, Isabelle
Dominguez, Natalia
Abrégé
The invention relates to a peptide-modified AAV capsid with improved tropism, in particular increased muscle tropism and/or reduced liver tropism. The invention relates also to the derived recombinant AAV vector particle packaging a gene of interest, and its use in gene therapy, in particular for treating muscle diseases.
LSD1 INHIBITOR AND PRMT6 INHIBITOR FOR USE IN THE TREATMENT OF A DISEASE ASSOCIATED WITH GAIN-OF-FUNCTION OF ANDROGEN RECEPTOR (AR) AND/OR WITH OVEREXPRESSION OF AN AR COACTIVATOR
FONDAZIONE ISTITUTO ITALIANO DI TECNOLOGIA (Italie)
GENETHON (France)
UNIVERSITA' DEGLI STUDI DI TRENTO (Italie)
INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE (INSERM) (France)
UNIVERSITÉ D'ÉVRY-VAL D'ESSONNE (France)
UNIVERSITA' DEGLI STUDI DI PADOVA (Italie)
Inventeur(s)
Pennuto, Maria
Basso, Manuela
Ronzitti, Giuseppe
Contestabile, Andrea
Abrégé
The present invention is directed to a therapeutic agent for use in the treatment of a disease associated with gain-of-function of androgen receptor (AR) and/or with overexpression of an AR coactivator, such as Kennedy disease or cancer. The therapeutic agent of the invention comprises at least one inhibitor of an androgen receptor (AR) coactivator, selected from protein arginine methyltransferase 6 (PRMT6) inhibitor, lysine specific demethylase 1 (LSD1) inhibitor, or combinations thereof.
A61K 31/7105 - Acides ribonucléiques naturels, c.-à-d. contenant uniquement des riboses liés à l'adénine, la guanine, la cytosine ou l'uracile et ayant des liaisons 3'-5' phosphodiester
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE (France)
Inventeur(s)
Cosette, Jérémie
Stockholm, Daniel
Abrégé
The invention relates to a method for predicting treatment efficiency in the research phase. The steps of the method comprise detecting at least one biological parameter in a histological section image; determining a mosaic of the image (squares or rectangles, also referred to as tiles); defining an image with smaller dimensions (also referred to as a density map) by integrating marked biological parameters in each fraction of the mosaic; applying deep learning algorithms, which are implemented, for example, by a convolutional neural network on the images generated in step c. in order to determine a proximity score for the images of treated tissues with respect to images of healthy tissues or untreated diseased tissues.
G06V 10/82 - Dispositions pour la reconnaissance ou la compréhension d’images ou de vidéos utilisant la reconnaissance de formes ou l’apprentissage automatique utilisant les réseaux neuronaux
28.
HYBRID AAV VECTOR ENHANCING TRANSGENE EXPRESSION IN THE LIVER
INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE (France)
UNIVERSITE EVRY VAL D'ESSONNE (France)
Inventeur(s)
La Bella, Tiziana
Ronzitti, Giuseppe
Siauve, Justine
Abrégé
The invention relates to a hybrid AAV vector comprising a transgene of interest operably linked to an hepatocyte-specific promoter, which enhances transgene expression in the liver and its use for liver-directed gene therapy.
INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE (France)
UNIVERSITE EVRY VAL D'ESSONNE (France)
Inventeur(s)
Galy, Anne
Richard, Isabelle
Rocca, Céline
Albini, Sonia
Ferrand, Maxime
Abrégé
The invention relates to an immune cell engineered to express a chimeric antigen receptor (CAR) which specifically binds Fibroblast Activation Protein (FAP) for use in the treatment of skeletal muscle fibrosis in muscular dystrophies.
C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
C07K 16/40 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre des enzymes
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
05 - Produits pharmaceutiques, vétérinaires et hygièniques
09 - Appareils et instruments scientifiques et électriques
10 - Appareils et instruments médicaux
35 - Publicité; Affaires commerciales
36 - Services financiers, assurances et affaires immobilières
41 - Éducation, divertissements, activités sportives et culturelles
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Pharmaceutical, veterinary and hygienic products; chemical
preparations for medical and pharmaceutical use or for
veterinary use; biological preparations for medical and
pharmaceutical use or for veterinary use; enzyme
preparations for medical and pharmaceutical use or for
veterinary use; microorganism cultures for medical and
pharmaceutical use or for veterinary use; culture media for
bacteriology; nutritional substances for microorganisms;
preparations of micro-organisms, of genetically modified
organisms and of cells for medical and pharmaceutical use or
for veterinary use, particularly for gene therapy and cell
therapy; compounds acting on genetic or other pathologies,
in human beings and animals, particularly on muscle and
neuromuscular diseases, diseases of the immune system,
cardiovascular diseases, dermatological diseases, diseases
of the central and peripheral nervous system, cancers,
asthma, allergies, lipid disorders, obesity and diabetes and
infectious diseases; kits for preparing cells or for
selecting biologically active compounds; diagnostic products
for medical or veterinary use; diagnostic reagents for
medical or veterinary use; diagnostic reagents for in-vitro
use in biochemistry, clinical chemistry and microbiology;
hygienic products for medical use; diagnostic biomarkers for
use in neuromuscular clinical research for medical use. Scientific and research apparatus and instruments; apparatus
and instruments for recording, transmitting, reproducing or
processing sound, images or data; patient (electronic)
database; software; mobile applications; downloadable
Software as a Medical Device [SaMD]; downloadable computer
programs; computer platforms in the form of recorded or
downloadable software. Surgical, medical apparatus and instruments; testing
apparatus for medical use; diagnostic apparatus for medical
use; cell culture instruments for medical use; surgical,
medical apparatus and instruments intended for use in gene
therapy and cell therapy; medical devices. Business management assistance; commercial business
appraisals; business management and organization
consultancy; business management consultancy; business
expertise; business information; business inquiries;
commercial administration of the licensing of third-party
goods and services; commercial or industrial company
management assistance; advice on development strategy for
scientific and technology projects (business management
assistance); administrative management of contracts relating
to research; price comparison services; establishing
statistics; compilation of information into computer
databases; systematization of data in a central file;
computer file management; market research and studies;
organization of fairs, exhibitions, trade fairs and job
fairs for commercial or advertising purposes; searching for
industrial or commercial partners; searching for financial
partners; economic forecasting; sales promotion for others;
advertising; commercial administration, commercial
management advice, marketing advice, commercial strategy
advice, business management advice, as well as office
functions for the development and innovation of companies in
the health sector; commercial feasibility studies, business
appraisals, marketing studies, accounting, economic
forecasting, and searching for sponsors to implement
development plans for companies in the health sector;
computerized file management for streamlining the business
branches of companies in the health sector; administrative
and commercial management and creation of services enabling
the organization of networks and matching partners and/or
service providers in areas of technological, innovation
expertise, financing various projects; database management;
compilation of information in computer databases;
consultancy and advisory services with respect to company
creation; advice and assistance to companies, particularly
in the context of a program for the selection, coaching and
acceleration of projects; sponsorship search; company audits
(commercial analyses). Providing financing for inventions; financing services in
the field of health; financial analysis; financial
appraisal; providing financial information; administration
and management of grants for medical research; providing
financial subsidies; providing project grants for health
awareness projects; financial investments; investment
research; advice with respect to financing and investment;
fundraising services; raising or investing capital; fund
investment. Education; training; entertainment; sporting and cultural
activities; information relating to entertainment or
education; publication of books; organization of
competitions (education or entertainment); organization of
exhibitions for cultural or educational purposes; electronic
publication of books and journals online; electronic desktop
publishing; organizing and conducting training workshops,
colloquiums, conferences, congresses, seminars, symposiums
for cultural or educational purposes; planning of receptions
(entertainment); drafting and publication of texts other
than advertising texts; publishing the results of clinical
trials conducted on patients and scientific articles in
relation to clinical and genetic diseases; providing
non-downloadable electronic publications from a global
computer network or the Internet; publishing services for
magazines in paper and in electronic form. Evaluations, assessments and research in the fields of
science and technology provided by engineers; appraisals and
assessments in the field of medical research; research in
the medical field; industrial analysis and research services
in the health sector; research and development of new
products (for others); technical project studies; scientific
research for medical purposes; clinical trials; medical
research laboratory services; biological, bacteriological,
gene and cell therapy, chemical and genetic research;
programming of software for Internet platforms; hosting of
platforms on the Internet; development of databases;
providing imaging and physiology platforms; research and
development of pharmaceutical, veterinary and sanitary
products for third parties; sorting and selection of
molecules; developing and validating biological tests, cell
tests, genetic tests and immunological tests; fundamental
and applied research in the scientific, technological or
industrial field; technical research; activities in
functional genomics for identifying, characterizing,
validating or developing biologically active molecules;
technical project study; professional consultancy unrelated
to business operations in the field of scientific research
and therapies. Medical and surgical services; medical and surgical
diagnosis; medical testing; hygienic and beauty care for
human beings; health service; advice with respect to health;
medical assistance; hospital services; nursing homes;
convalescence or rest homes; consultation with respect to
pharmacy; provision of information in the pharmaceutical
field; medical imaging services; human tissue bank services;
behavioral analysis for medical purposes; providing medical
information; conducting medical examinations; medical
consultation services; medical testing for the diagnosis and
treatment of persons; genetic testing services for medical
purposes; telemedicine services; therapeutic services.
INTERNATIONAL CENTRE FOR GENETIC ENGINEERING AND BIOTECHNOLOGY (Italie)
Inventeur(s)
Mingozzi, Federico
Ronzitti, Giuseppe
Collaud, Fanny
Muro, Andrés
Bortolussi, Giulia
Abrégé
The invention relates to a nucleic acid sequence useful in the treatment of hyperbilirubinemia, in particular in the treatment of Crigler-Najjar syndrome. More particularly, the nucleic acid sequence of the present invention is a codon-optimized UGT1A1 coding sequence.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 9/44 - Hydrolases (3.) agissant sur les composés glycosyliques (3.2) agissant sur les liaisons alpha-glucosidiques-1, 6, p. ex. iso-amylase, pullulanase
A61K 38/00 - Préparations médicinales contenant des peptides
34.
PEPTIDE-MODIFIED AAV CAPSID WITH ENHANCED MUSCLE TRANSDUCTION EFFICIENCY
INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE (France)
UNIVERSITE D'EVRY VAL D'ESSONNE (France)
Inventeur(s)
Richard, Isabelle
Hong, Ai Vu
Abrégé
The invention relates to a peptide-modified AAV capsid having an increased muscle transduction efficiency. The invention relates also to the derived recombinant AAV vector particle packaging a gene of interest, and its use in gene therapy, in particular for treating muscle diseases.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
09 - Appareils et instruments scientifiques et électriques
10 - Appareils et instruments médicaux
35 - Publicité; Affaires commerciales
36 - Services financiers, assurances et affaires immobilières
41 - Éducation, divertissements, activités sportives et culturelles
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
(1) Produits pharmaceutiques, vétérinaires et hygiéniques; préparations chimiques à usage médical, pharmaceutique ou à usage vétérinaire; préparations biologiques à usage médical, pharmaceutique ou à usage vétérinaire; préparations enzymatiques à usage médical, pharmaceutique ou à usage vétérinaire; cultures de micro-organismes à usage médical, pharmaceutique ou vétérinaire; bouillons de culture pour la bactériologie; substances nutritives pour micro-organismes; préparations de micro-organismes, d'organismes génétiquement modifiées et de cellules à usage médical, pharmaceutique ou à usage vétérinaire, en particulier pour la thérapie génique et la thérapie cellulaire; composés actifs sur les pathologies génétiques ou non, des êtres humains et des animaux, notamment sur les maladies musculaires, neuromusculaires, du système immunitaire, cardio-vasculaires, les maladies dermatologiques, les maladies du système nerveux central et périphérique, les cancers, l'asthme, les allergies, les désordres lipidiques, l'obésité et le diabète, les maladies infectieuses; kits pour la préparation de cellules ou pour la sélection de composés biologiquement actifs; produits pour le diagnostic à usage médical ou vétérinaire; réactifs pour le diagnostic à usage médical ou vétérinaire; réactifs pour le diagnostic pour un usage in vitro en biochimie, chimie clinique et en microbiologie; produits hygiéniques à usage médical; biomarqueurs de diagnostic pour la recherche clinique neuromusculaire à usage médical.
(2) Appareils et instruments scientifiques, de recherche; appareils et instruments pour l'enregistrement, la transmission, la reproduction ou le traitement de sons, d'images ou de données; base de données (électronique) de patients; logiciels; applications mobiles; logiciels en tant que dispositifs médicaux [SaMD] téléchargeables; programmes informatiques téléchargeables; plateformes informatiques sous forme de logiciels enregistrés ou téléchargeables.
(3) Appareils et instruments chirurgicaux, médicaux; appareils pour l'analyse à usage médical; appareils pour le diagnostic à usage médical; instruments de culture cellulaire à usage médical; appareils et instruments chirurgicaux, médicaux destinés à la thérapie génique et la thérapie cellulaire; dispositifs médicaux. (1) Aide à la direction des affaires; estimations en affaires commerciales; conseils en organisation et direction des affaires; consultations pour la direction des affaires; expertises en affaires; informations d'affaires; renseignements d'affaires; administration commerciale de licences de produits et de services de tiers; aide à la direction d'entreprises industrielles ou commerciales; conseil en stratégie de développement de projets scientifiques et technologie (aide à la direction des affaires); gestion administrative de contrats en lien avec la recherche; services de comparaison de prix; établissement de statistiques; recueils de données dans un fichier central; systématisation de données dans un fichier central; gestion de fichiers informatiques; études et recherches de marchés; organisation de salons, d'expositions, de foires à buts commerciaux ou de publicité; recherche de partenaires industriels ou commerciaux; recherche de partenaires financiers; prévisions économiques; promotions des ventes pour des tiers; publicité; administration commerciale, prestations de conseils en gestion commerciale, prestations de conseils en marketing, prestations de conseil en stratégie commerciale, prestations de conseils en direction des affaires, ainsi que travaux de bureau pour le développement et l'innovation des entreprises dans le secteur de la santé; étude de faisabilité commerciale, estimation d'affaire, étude de marché, comptabilité, prévisions économiques, et recherche de parraineurs pour la réalisation de plans de développement à destination des entreprises du secteur de la santé; gestion de fichiers informatiques pour la rationalisation des branches d'activité des entreprises du secteur de la santé; services de gestion et de création administrative et commerciale de prestations permettant l'organisation de réseaux et de mise en contact de partenaires et/ou prestataires sur des pôles de compétences technologiques, d'innovation, de financement de projets divers; gestion de bases de données; services de compilation de données dans des bases de données informatiques; services de consultations et de conseils en matière de création d'entreprise; services de conseils et d'aide aux entreprises, notamment dans le cadre d'un programme de sélection, d'accompagnement et d'accélération de projets; recherche de parraineurs; audits d'entreprises (analyses commerciales).
(2) Mise à disposition de financement pour des inventions; services de financement dans le domaine de la santé; analyse financière; estimation financière; mise à disposition d'informations financières; administration et gestion de subventions pour la recherche médicale; mise à disposition de subventions financières; mise à disposition de subventions de projets dans le cadre de projets de sensibilisation aux questions de santé; investissements financiers; recherche en investissement; conseils en matière de financement et d'investissement; collectes de fonds; constitution ou investissement de capitaux; placement de fonds.
(3) Éducation; formation; divertissement; activités sportives et culturelles; informations en matière de divertissement ou d'éducation; publication de livres; organisation de concours (éducation ou divertissement); organisation d'expositions à buts culturels ou éducatifs; publication électronique de livres et de périodiques en ligne; micro-édition; organisation et conduite d'ateliers de formation, de colloques, de conférences, de congrès, de séminaires, de symposiums à des fins culturelles ou éducatives; planification de réceptions (divertissement); rédaction et publication de textes autres que textes publicitaires; publication de résultats d'essais cliniques réalisés sur des malades et d'articles scientifiques en rapport avec les maladies cliniques et génétiques; mise à disposition de publications électroniques, non téléchargeables, à partir d'un réseau informatique mondial ou d'Internet; services de publication de magazines papiers et sous forme électronique.
(4) Evaluations, estimations et recherches dans les domaines scientifique et technologique rendues par des ingénieurs; évaluations et estimations dans le domaine de la recherche médicale; recherches dans le domaine médical; services d'analyse et de recherches industrielles dans le secteur de la santé; recherche et développement de nouveaux produits (pour des tiers); études de projets techniques; recherches scientifiques à but médical; essais cliniques; services de laboratoire de recherche médicale; recherches biologiques, en bactériologie, en thérapie génique et cellulaire, en chimie et en génétique; programmation de logiciels pour des plateformes Internet; hébergement de plateformes sur Internet; développement de bases de données; mise à disposition de plateformes d'imagerie et de physiologie; services de recherche et développement de produits pharmaceutiques, vétérinaires et hygiéniques pour des tiers; triage et sélection de molécules; développement et validation de tests biologiques, de tests cellulaires, de tests génétiques et de tests immunologiques; recherche fondamentale et appliquée dans le domaine scientifique, technologique ou industrielle; recherches techniques; activités de génomique fonctionnelle, pour identifier, caractériser, valider ou développer des molécules biologiquement actives; étude de projets techniques; consultations professionnelles sans rapport avec la conduite des affaires dans le domaine de la recherche scientifique et des thérapies.
(5) Services médicaux et chirurgicaux; diagnostic médical et chirurgical; tests médicaux; soins d'hygiène et de beauté pour êtres humains; service de santé; conseils en matière de santé; assistance médicale; services hospitaliers; maisons médicalisées; maisons de convalescence ou de repos; consultation en matière de pharmacie; mise à disposition d'informations dans le domaine pharmaceutique; services d'imagerie médicale; services de banques de tissus humains; analyses comportementales à des fins médicales; mise à disposition d'informations médicales; réalisation d'examens médicaux; services de consultations médicales; analyses médicales pour le diagnostic et le traitement de personnes; services de dépistage génétique à des fins médicales; services de télémédecine; services thérapeutiques.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
09 - Appareils et instruments scientifiques et électriques
10 - Appareils et instruments médicaux
35 - Publicité; Affaires commerciales
36 - Services financiers, assurances et affaires immobilières
41 - Éducation, divertissements, activités sportives et culturelles
42 - Services scientifiques, technologiques et industriels, recherche et conception
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Pharmaceutical, veterinary products, namely gene and cell therapy products for the treatment of diseases in the nature of gene therapy preparations and genetically engineered pharmaceutical preparations for the treatment of diseases and disorders of genetic etiology; chemical preparations for medical and pharmaceutical use or for veterinary use, namely gene and cell therapy products for the treatment of diseases; biological preparations for medical and pharmaceutical use or for veterinary use, namely gene and cell therapy products for the treatment of diseases; enzyme preparations for medical and pharmaceutical use or for veterinary use, namely gene and cell therapy products for the treatment of diseases; microorganism cultures for medical and pharmaceutical use or for veterinary use; culture media for bacteriology; nutritional substances for microorganisms; preparations of micro-organisms, of genetically modified organisms and of cells for medical and pharmaceutical use or for veterinary use, particularly for gene therapy and cell therapy; compounds acting on genetic or other pathologies, in human beings and animals, particularly on muscle and neuromuscular diseases, diseases of the immune system, cardiovascular diseases, dermatological diseases, diseases of the central and peripheral nervous system, cancers, asthma, allergies, lipid disorders, obesity and diabetes and infectious diseases; kits for preparing cells or for selecting biologically active compounds comprised of gene therapies, namely biologically active cell preparations for the treatment of diseases and disorders of genetic etiology; diagnostic products for medical or veterinary use; diagnostic reagents for medical or veterinary use; diagnostic reagents for in-vitro use in biochemistry, clinical chemistry and microbiology; diagnostic biomarkers for use in neuromuscular clinical research for medical use Electronic database in the field of medical patients; downloadable Software for artificial intelligence and machine learning software; downloadable mobile applications for artificial intelligence and machine learning software; downloadable Software as a Medical Device [SaMD]; downloadable computer programs for artificial intelligence and machine learning software; Computer platforms in the form of recorded or downloadable software for artificial intelligence and machine learning software Surgical, medical apparatus and instruments for medical use; testing apparatus for medical use for the diagnosis and/or follow-up the evolution of diseases namely genetic diseases; Diagnostic apparatus for medical use for the diagnosis and/or follow-up the evolution of diseases namely genetic diseases; cell culture instruments for medical use; surgical, medical apparatus and instruments intended for use in gene therapy and cell therapy; medical devices namely medical, biological and gene therapy testing kits to assist in collecting, assessing and determining optimal drugs and biological products comprising specimen transport tubes for use with blood, genetic materials, fluids, tissues, samples and other human components and related elements, frozen specimen bags, specimen collection and transport instructions, foam coolers for specimens, emergency medical service foam coolers for specimens, cardboard shipping containers for specimens, and shipping labels for specimens, all for medical use; medical devices, namely active implantable medical devices namely implantable drug delivery pumps and implantable drug delivery ports Business management assistance; commercial business appraisals; business management and organization consultancy; business management consultancy; Business expertise, in particular for start-up creation, mentoring programs; business information; business inquiries; commercial administration of the licensing of third-party goods and services; commercial or industrial company management assistance; advice on development strategy for scientific and technology projects (business management assistance); administrative management of contracts relating to research; price comparison services; establishing statistics; compilation of information into computer databases; systematization of data in a central file; computer file management; market research and studies; organization of fairs, exhibitions, trade fairs and job fairs for commercial or advertising purposes; searching for industrial or commercial partners; searching for financial partners; economic forecasting; sales promotion for others; advertising; commercial administration, commercial management advice, marketing advice, commercial strategy advice, business management advice, as well as office functions for the development and innovation of companies in the health sector; commercial feasibility studies, business appraisals, marketing studies, accounting, economic forecasting, and searching for sponsors to implement development plans for companies in the health sector; computerized file management for streamlining the business branches of companies in the health sector; administrative and commercial management and creation of services enabling the organization of networks and matching partners and/or service providers in areas of technological, innovation expertise, financing various projects; database management; compilation of information in computer databases; consultancy and advisory services with respect to company creation; advice and assistance to companies, particularly in the context of a program for the selection, coaching and acceleration of projects; sponsorship search; company audits (commercial analyses) Providing financing for inventions through, in particular, public financing ; financing services in the field of health; financial analysis; Financing appraisal through monitoring of use of public financing; providing financial information; administration and management of grants for medical research; providing financial subsidies; providing project grants for health awareness projects; financial investments; Investment research through, in particular, public financing; advice with respect to financing and investment; fundraising services; raising or investing capital; fund investment Education in the nature of digital training platforms, development of training digital content, photography and video for education purposes; training in the nature of organization of exhibitions for educational purposes, arranging and conducting of conferences, congresses and symposiums, publication of content; information relating to entertainment or education; publication of books; organization of competitions (education or entertainment); organization of exhibitions for cultural or educational purposes; electronic publication of books and journals online; electronic desktop publishing; organizing and conducting training workshops, colloquiums, conferences, congresses, seminars, symposiums for cultural or educational purposes; planning of receptions (entertainment); drafting and publication of texts other than advertising texts; publishing the results of clinical trials conducted on patients and scientific articles in relation to clinical and genetic diseases; providing non-downloadable electronic publications from a global computer network or the Internet; publishing services for magazines in paper and in electronic form Evaluations, assessments and research in the fields of science and technology provided by engineers; appraisals and assessments in the field of medical research; research in the medical field; industrial analysis and research services in the health sector; research and development of new products (for others); technical project studies; scientific research for medical purposes; clinical trials; medical research laboratory services; biological, bacteriological, gene and cell therapy, chemical and genetic research; programming of software for Internet platforms; hosting of platforms on the Internet; development of databases; providing imaging and physiology platforms; research and development of pharmaceutical, veterinary and sanitary products for third parties; sorting and selection of molecules; developing and validating biological tests, cell tests, genetic tests and immunological tests; fundamental and applied research in the scientific, technological or industrial field; technical research; activities in functional genomics for identifying, characterizing, validating or developing biologically active molecules; technical project study; professional consultancy unrelated to business operations in the field of scientific research and therapies Medical and surgical services; medical and surgical diagnosis; medical testing; hygienic and beauty care for human beings; health service; advice with respect to health; medical assistance; hospital services; nursing homes; convalescence or rest homes; consultation with respect to pharmacy; provision of information in the pharmaceutical field; medical imaging services; human tissue bank services; behavioral analysis for medical purposes; providing medical information; conducting medical examinations; medical consultation services; medical testing for the diagnosis and treatment of persons; genetic testing services for medical purposes; telemedicine services
37.
CNS gene delivery using peripheral administration of AAV vectors
CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (France)
Inventeur(s)
Barkats, Martine
Abrégé
The present invention relates to compositions and methods for the delivery of therapeutic proteins to the CNS using recombinant AAV vectors. More specifically, the invention relates to compositions and methods for delivering proteins into the cerebrospinal fluid of mammalian subjects through peripheral administration of AAV vectors. The invention may be used to treat various disorders of the central nervous system, including degenerative diseases and motor neuron diseases.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 25/00 - Médicaments pour le traitement des troubles du système nerveux
38.
COMBINATION OF A PROTEASOME INHIBITOR AND A HDAC INHIBITOR AND ITS USE FOR THE TREATMENT OF GENETIC DISEASES LINKED TO A PROTEIN CONFORMATIONAL DISORDER
Centre d'Etude des Cellules Souches (CECS) (France)
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (France)
Genethon (France)
UNIVERSITE D'EVRY-VAL-D'ESSONNE (France)
Inventeur(s)
Hoch, Lucile
Nissan, Xavier
Richard, Isabelle
Abrégé
The present invention relates to the combination of a proteasome inhibitor and a histone deacetylase (HDAC) inhibitor and its use for the treatment of a genetic disease linked to a conformational disorder of at least one protein, said disorder causing the proteasome degradation of the protein.
A61K 31/5355 - Oxazines non condensées contenant d'autres hétérocycles
A61K 31/167 - Amides, p. ex. acides hydroxamiques ayant des cycles aromatiques, p. ex. colchicine, aténolol, progabide ayant l'atome d'azote d'un groupe carboxamide lié directement au cycle aromatique, p. ex. lidocaïne, paracétamol
Inserm (Institut National De La Sante Et De La Recherche Medicale) (France)
Inventeur(s)
Richard, Isabelle
Gicquel-Zouida, Evelyne
Lostal, William
Abrégé
The present invention concerns synthetic polynucleotides encoding a human fukutin-related protein (FKRP) wherein the synthetic polynucleotides contain at least a mutation avoiding supplementary transcript(s) generated from frameshift start codon(s). The synthetic polynucleotides are useful, especially for treating a pathology linked to a FKRP deficiency or induced by a defect in α-dystroglycan (α-DG) glycosylation, such as LGMD2I.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
CENTRE D'ETUDE DES CELLULES SOUCHES (CECS) (France)
GENETHON (France)
UNIVERSITE D'EVRY-VAL-D'ESSONNE (France)
INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) (France)
Inventeur(s)
Nissan, Xavier
Bruge, Céline
Pellier, Emilie
Richard, Isabelle
Abrégé
The invention relates to the use of bazedoxifene as representing third-generation SERM, namely compounds comprising a 2-phenylindole group, for increasing muscle survival, in particular in the treatment of dysferlinopathies.
A61K 31/55 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p. ex. guanéthidine ou rifamycines ayant des cycles à sept chaînons, p. ex. azélastine, pentylènetétrazole
A61K 31/454 - Pipéridines non condensées, p. ex. pipérocaïne contenant d'autres systèmes hétérocycliques contenant un cycle à cinq chaînons avec l'azote comme hétéro-atome du cycle, p. ex. pimozide, dompéridone
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCH MEDICALE) (France)
UNIVERSITE D'EVRY-VAL-D'ESSONNE (France)
Inventeur(s)
Lostal, William
Richard, Isabelle
Abrégé
The present invention concerns a novel short promoter characterized by a high activity in the skeletal muscles and a low activity in the heart. It then constitutes a valuable candidate especially for driving the expression of transgenes encoding proteins useful for the treatment of muscular dystrophies.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 9/64 - Protéinases provenant de tissu animal, p. ex. rennine
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
Institut National de la Santé et de la Recherche Médicale (France)
Inventeur(s)
Richard, Isabelle
Biquand, Ariane
Abrégé
The invention relates to the treatment of dilated cardiomyopathies, in particular genetic dilated cardiomyopathies, using non-expressible inhibitors of the WNT pathway or TGF-β pathway, alone or in combination.
A61K 31/519 - PyrimidinesPyrimidines hydrogénées, p. ex. triméthoprime condensées en ortho ou en péri avec des hétérocycles
A61K 31/4439 - Pyridines non condenséesLeurs dérivés hydrogénés contenant d'autres systèmes hétérocycliques contenant un cycle à cinq chaînons avec l'azote comme hétéro-atome du cycle, p. ex. oméprazole
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
A61P 9/00 - Médicaments pour le traitement des troubles du système cardiovasculaire
44.
GENE THERAPY EXPRESSION SYSTEM ALLEVIATING CARDIAC TOXICITY OF FKRP
INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) (France)
Inventeur(s)
Gicquel, Evelyne
Richard, Isabelle
Abrégé
The present invention concerns an expression system for systemic administration comprising a sequence encoding a FKRP protein, and:
a promoter sequence allowing the expression at a therapeutically acceptable level of FKRP in the skeletal muscles and a target sequence of an miRNA expressed in the heart; or
a promoter sequence allowing the expression at a therapeutically acceptable level of FKRP in the skeletal muscles and presenting a promoter activity at a toxically acceptable level in the heart;
and its use for the treatment of various diseases linked to FKRP deficiencies.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
45.
Enhancing Utrophin Expression in Cell by Inducing Mutations Within Utrophin Regulatory Elements and Therapeutic Use Thereof
Institut National de la Santé et de la Recherche Médicale (France)
Inventeur(s)
Amendola, Mario
Guiraud, Simon
Abrégé
The invention relates to a composition for enhancing utrophin expression in cell by inducing mutations within a target sequence comprising a utrophin repressor binding site using a gene editing enzyme and the use thereof for the treatment of a dy-strophinopathy.
Institut National de la Santé et de la Recherche Médicale (France)
Universite d'Evry Val d'Essonne (France)
Inventeur(s)
Richard, Isabelle
Biquand, Ariane
Abrégé
The invention relates to the treatment of genetic dilated cardiomyopathies using expressible modulators of the Wnt pathway or TGF-β pathway, preferably using gene transfer.
INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) (France)
UNIVERSITE D’EVRY-VAL-D’ESSONNE (France)
Inventeur(s)
Richard, Isabelle
Poupiot, Jerome
Abrégé
The present invention concerns an expression system for systemic administration comprising a sequence encoding gamma-sarcoglycan (SGCG) placed under the control of a promoter allowing an adequate expression of SGCG in the skeletal muscles and in the heart, and its use for the treatment of Limb-Girdle Muscular Dystrophy type C.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
48.
NRF2 Activator for Use in Treating Dilated Cardiomyopathies
A61P 9/10 - Médicaments pour le traitement des troubles du système cardiovasculaire des maladies ischémiques ou athéroscléreuses, p. ex. médicaments antiangineux, vasodilatateurs coronariens, médicaments pour le traitement de l'infarctus du myocarde, de la rétinopathie, de l'insuffisance cérébro-vasculaire, de l'artériosclérose rénale
A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
49.
Method for Engineering Novel Hybrid AAV Capsids Through Hyper Variable Regions Swapping
Institut National de la Santé et de la Recherche Médicale (France)
Universite d'Evry Val d'Essonne (France)
Inventeur(s)
Ronzitti, Giuseppe
Labella, Tiziana
Abrégé
The invention relates to a method of preparation of a recombinant hybrid adeno-associated virus (AAV) capsid protein with improved tropism and to the recombinant hybrid AAV capsid protein obtainable by the method. The invention relates also to the derived expression vector, modified cell, and hybrid capsid AAV vector particle packaging a gene of interest, and its use in tissue-targeted gene therapy for treating various diseases.
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) (France)
UNIVERSITE D'EVRY VAL D'ESSONNE (France)
UNIVERSITE CLAUDE BERNARD LYON 1 (France)
Inventeur(s)
Jauze, Louisa
Rajas, Fabienne
Ronzitti, Giuseppe
Abrégé
The invention relates to an adeno-associated virus (AAV) vector comprising a nucleic acid construct for the expression of a glucose-6-phosphatase-a (G6Pase-a) in a cell, the construct comprising a nucleic acid sequence encoding the G6Pase-a, wherein the nucleic acid sequence encoding the G6Pase-a is operably linked to a human alpha-1 antitrypsin (hAAT) promoter, a cell transformed with the vector of the invention, a composition comprising the vector or the cell of the invention, and the use thereof.
INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÈDICAL) (France)
Inventeur(s)
Galy, Anne
Abrégé
The present invention relates to a method for obtaining stable pseudotyped lentiviral particles including a heterologous gene of interest, comprising the following steps:
a) transfecting at least one plasmid in appropriate cell lines, wherein said at least one plasmid comprises the gene of interest, the rev, gag and pol genes, and a sequence coding for an ERV syncytin, wherein the rev, gag and pol genes are retroviral genes;
b) incubating the transfected cells obtained in a), so that they produce the stable pseudotyped lentiviral particles in the supernatant; and
c) harvesting and concentrating the stable lentiviral particles obtained in b).
The present invention relates to a method for obtaining stable pseudotyped lentiviral particles including a heterologous gene of interest, comprising the following steps:
a) transfecting at least one plasmid in appropriate cell lines, wherein said at least one plasmid comprises the gene of interest, the rev, gag and pol genes, and a sequence coding for an ERV syncytin, wherein the rev, gag and pol genes are retroviral genes;
b) incubating the transfected cells obtained in a), so that they produce the stable pseudotyped lentiviral particles in the supernatant; and
c) harvesting and concentrating the stable lentiviral particles obtained in b).
The present invention also relates to a method to transduce immune cells using lentiviral vectors pseudotyped with an ERV syncytin glycoprotein. The method can be performed on non-stimulated blood cells or on cells stimulated briefly with IL7, and the cells can be expanded.
The present invention relates to a method for obtaining stable pseudotyped lentiviral particles including a heterologous gene of interest, comprising the following steps:
a) transfecting at least one plasmid in appropriate cell lines, wherein said at least one plasmid comprises the gene of interest, the rev, gag and pol genes, and a sequence coding for an ERV syncytin, wherein the rev, gag and pol genes are retroviral genes;
b) incubating the transfected cells obtained in a), so that they produce the stable pseudotyped lentiviral particles in the supernatant; and
c) harvesting and concentrating the stable lentiviral particles obtained in b).
The present invention also relates to a method to transduce immune cells using lentiviral vectors pseudotyped with an ERV syncytin glycoprotein. The method can be performed on non-stimulated blood cells or on cells stimulated briefly with IL7, and the cells can be expanded.
The stable pseudotyped lentiviral particles obtained are particularly useful in gene therapy.
INSERM (Institut National de la Santé et de la Recherche Médicale) (France)
Genethon (France)
Université d'Evry-Val-d'Essonne (France)
Inventeur(s)
Sylvain, Fisson
Abrégé
Despite the eye's immune-privileged status, a secondary loss of vision in some patients treated with AAV led the inventors to question the immunogenicity of AAV vectors after a subretinal injection. The inventors thus characterized anti-transgene and anti-capsid immune responses induced in the periphery after the subretinal AAV injection. Different doses of AAV8 encoding reporter proteins fused with the HY male antigen were injected at day 0 into the subretinal space of adult immunocompetent C57BL/6 female mice. Subretinal AAV injection induced a dose-dependent proinflammatory immune response to the transgene product, correlated with local transgene expression. In order to trigger a subretinal-associated immune inhibition (SRAII) mechanism, some mice were co-injected subretinally at day 0 with AAV and HY peptides. Interestingly, this subretinal co-injection of AAV8 with peptides of the transgene product modulated the anti-transgene T-cell immune response, even at high dose of vector (5.1010 vg). This immunodulation was also confirmed in a pathophysiological murine model of retinal degeneration. The inventors also demonstrated that injection of AAV8 in the subretinal space induces proinflammatory peripheral immune responses to the transgene and the capsid that could be counteracted y co-injection with transgene peptides. Accordingly, the object of the present invention is to provide methods for preventing induction of immune responses to the transgene product and the AAV capsid after ocular gene therapy.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61K 38/16 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés
INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE (France)
UNIVERSITE D'EVRY VAL D’ESSONNE (France)
Inventeur(s)
Richard, Isabelle
Dorange, Fabien
Poupiot, Jérôme
Abrégé
The present invention relates to a gene expression construct comprising an expression cassette containing a transgene encoding a gamma-sarcoglycan protein and modified ITR sequences, said transgene encoding the gamma-sarcoglycan protein consisting of the sequence of SEQ ID NO: 3 or a functional variant thereof having a polynucleotide sequence that is at least 80% identical to SEQ ID NO: 3; said modified ITR sequences consisting of - a modified 5'-ITR sequence of SEQ ID NO: 1; and - a modified 3'-ITR sequence of SEQ ID NO: 2. The invention also relates to a recombinant vector comprising the gene expression construct of the invention, said gene expression construct or recombinant vector can be included in a gene therapy and used as a medicament, in particular for the treatment of limb girdle muscular dystrophy by sarcoglycanopathy.
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
55.
CALPAIN-3 GENE TRANSFER INCREASE USING MODIFIED ITR SEQUENCES
INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE (France)
UNIVERSITE D'EVRY VAL D’ESSONNE (France)
Inventeur(s)
Richard, Isabelle
Dorange, Fabien
Abrégé
The present invention relates to a gene expression construct comprising an expression cassette containing a transgene encoding a calpain-3 protein and modified ITR sequences, said transgene encoding the calpain-3 protein consisting of the sequence of SEQ ID NO: 4 or a functional variant thereof having a polynucleotide sequence that is at least 80% identical to SEQ ID NO: 4; said modified ITR sequences consisting of - a modified 5'-ITR sequence of SEQ ID NO: 1; and - a modified 3'-ITR sequence of SEQ ID NO: 2 or SEQ ID NO: 3. The invention also relates to a recombinant vector comprising the gene expression construct of the invention, said gene expression construct or recombinant vector can be included in a gene therapy and used as a medicament, in particular for the treatment of limb girdle muscular dystrophy by calpainopathy.
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 3/08 - Médicaments pour le traitement des troubles du métabolisme de l'homéostase du glucose
C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
C12N 9/16 - Hydrolases (3.) agissant sur les liaisons esters (3.1)
57.
ALPHA-SARCOGLYCAN GENE TRANSFER INCREASE USING MODIFIED ITR SEQUENCES
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (France)
UNIVERSITE D'EVRY VAL D’ESSONNE (France)
Inventeur(s)
Richard, Isabelle
Dorange, Fabien
Abrégé
The present invention relates to a gene expression construct comprising an expression cassette containing a transgene encoding an alpha-sarcoglycan protein and modified ITR sequences, said transgene encoding the alpha-sarcoglycan protein consisting of the sequence of SEQ ID NO: 3 or a functional variant thereof having a polynucleotide sequence that is at least 80% identical to SEQ ID NO: 3; said modified ITR sequences consisting of - a modified 5'-ITR sequence of SEQ ID NO: 1; and - a modified 3'-ITR sequence of SEQ ID NO: 2. The invention also relates to a recombinant vector comprising the gene expression construct of the invention, said gene expression construct or recombinant vector can be included in a gene therapy and used as a medicament, in particular for the treatment of limb girdle muscular dystrophy by sarcoglycanopathy.
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
58.
FUKUTIN RELATED PROTEIN GENE TRANSFER INCREASE USING MODIFIED ITR SEQUENCES
INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE (France)
UNIVERSITE D'EVRY VAL D’ESSONNE (France)
Inventeur(s)
Richard, Isabelle
Dorange, Fabien
Abrégé
The present invention relates to a gene expression construct comprising an expression cassette containing a transgene encoding a fukutin related protein and modified ITR sequences, said transgene encoding the fukutin related protein consisting of the sequence of SEQ ID NO: 3 or a functional variant thereof having a polynucleotide sequence that is at least 80% identical to SEQ ID NO: 3; said modified ITR sequences consisting of - a modified 5'-ITR sequence of SEQ ID NO: 1; and - a modified 3'-ITR sequence of SEQ ID NO: 2. The invention also relates to a recombinant vector comprising the gene expression construct of the invention, said gene expression construct or recombinant vector can be included in a gene therapy and used as a medicament, in particular for the treatment of limb girdle muscular dystrophy with FKRP deficiency.
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
59.
Use of a Synthetic AAV Capsid for Gene Therapy of Muscle and Central Nervous System Disorders
Institut National de la Santé et de la Recherche Médicale (France)
Inventeur(s)
Buj Bello, Ana
Renaud-Gabardos, Edith
Grimm, Dirk
Weinmann, Jonas
Abrégé
The invention relates to the use of a recombinant porcine adeno-associated virus (AAV) vector comprising a peptide-modified porcine AAV serotype 1 (AAVpol) capsid in gene therapy of muscle and/or central nervous system (CNS) disorders, in particular neuromuscular diseases such as genetic neuromuscular diseases.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
60.
VECTORS FOR THE TREATMENT OF ACID CERAMIDASE DEFICIENCY
Disclosed herein are methods for treating patients that may develop or already have pre-existing gene therapy neutralizing antibodies by administering a protease that cleaves peptide bonds present in immunoglobulins or by administering a glycosidase that cleaves carbohydrate residues present on immunoglobulins, or other similar enzymatic cleavage of immunoglobulins in vivo. Also disclosed are methods for utilizing IdeS and other immunoglobulin G-degrading enzyme polypeptides for gene therapy treatment of a disease in a patient in need thereof.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 9/24 - Hydrolases (3.) agissant sur les composés glycosyliques (3.2)
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
63.
HYBRID PROMOTERS FOR GENE EXPRESSION IN MUSCLES AND IN THE CNS
INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) (France)
Inventeur(s)
Ronzitti, Giuseppe
Vidal, Pauline
Abrégé
The present invention relates to novel hybrid promoters. The invention further relates to expression cassettes and vectors containing said hybrid promoters. Also disclosed herein are methods implementing these hybrid promoters, in particular methods of gene therapy.
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
The present invention relates to a method for producing a recombinant viral vector from hairy roots of a plant, in particular from hairy roots of a plant belonging to the Brassicaceae family. The invention also relates to a transgenic plant, a hairy root culture and a recombinant viral vector obtainable by the method of the invention.
INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) (France)
SORBONNE UNIVERSITE (France)
Inventeur(s)
Colella, Pasqualina
Puzzo, Francesco
Mingozzi, Federico
Abrégé
The present invention relates to a nucleic acid molecule encoding a chimeric polypeptide comprising a peptide of interest fused to one or more heterologous moieties, wherein at least one of the heterologous moieties is a ligand of the Sortilin receptor. The invention also relates to a chimeric polypeptide encoded by said nucleic acid molecule and uses thereof.
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) (France)
UNIVERSITE D'EVRY VAL D'ESSONNE (France)
Inventeur(s)
Mingozzi, Federico
Ronzitti, Giuseppe
Collaud, Fanny
Abrégé
The invention relates to a kit of parts comprising (i) pharmacological chaperones or a pharmaceutically acceptable salt thereof and (ii) a therapeutic acid-alpha glucosidase (GAA) polypeptide or a nucleic acid molecule encoding a therapeutic GAA polypeptide, wherein said pharmacological chaperones are 1-deoxynojirimycin (DNJ) or a derivative thereof and ambroxol (ABX) or a derivative thereof.
Institut National de la Santé et de la Recherche Médicale (France)
Assistance Publique-Hôpitaux de Paris (France)
Genethon (France)
Sorbonne Université (France)
Université d'Evry-Val-d'Essonne (France)
Universite Paris XIII Paris-Nord Villetaneuse (France)
Inventeur(s)
Zucman-Rossi, Jessica
Nault, Jean-Charles
La Bella, Tiziana
Ronzitti, Giuseppe
Imbeaud, Sandrine
Vidal, Patrice
Abrégé
Adeno-associated virus (AAV) is a defective mono-stranded DNA virus, endemic in human population (35-80%). Recurrent clonal AAV2 insertions are associated with the pathogenesis of rare human hepatocellular carcinoma (HCC) developed on normal liver. The aimed of the inventors was to characterize the natural history of AAV infection in the liver. Viral DNA was thus quantified in tumor and non-tumor liver tissues of 1461 patients. Presence of episomal form and viral mRNA expression were analyzed using a DNAse/TaqMan based assay and quantitative RT-PCR. In silico analyses using viral capture data explored viral variants and new clonal insertions. AAV DNA was detected in 21% of the patients equally distributed in 2 major viral subtypes: one similar to AAV2, the other hybrid between AAV2 and AAV13 sequences. Thus the inventors provided an integrated analysis of the wild type AAV infection in the liver with the identification of viral genotypes, molecular forms, helper virus relationship and viral integrations. These findings are important to understand wild type AAV biology and particularly relevant considering the large usage of AAV vector in liver-targeted gene therapy. Thus, the present invention relates to new adeno-associated virus (AAV) variants and uses thereof for gene therapy.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
The present invention relates to the industrialization of the production of recombinant lentiviral vectors in order to manufacture sufficient materials for therapeutic applications such as gene therapy and/or DNA vaccination, for use in clinical trials and/or commercial use.
CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (France)
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (France)
Inventeur(s)
Mingozzi, Federico
Davoust, Jean
Bartolo, Laurent
Abrégé
A combination of two recombinant adeno-associated viral (rAAV) vectors comprising in the first a capsid and a cassette comprising a 5′ ITR sequence, a liver-specific promotor, a nucleic acid sequence coding for a transgene of interest useful to be tolerated by the immune system and a poly A chain and in the second a capsid and a cassette comprising a 5′ ITR sequence, a promotor specific for a tissue of interest, a nucleic acid sequence corresponding to the nucleic acid sequence inserted into the cassette of the first rAAV vector, a transmembrane sequence, a poly A chain, wherein the nucleic acid sequences is administered towards to the tissue of interest, is disclosed. Said combination can be used in inducing an immune tolerance to a protein product encoded, and so be used as a drug in a subject, particularly for treating muscular dystrophies. Pharmaceutical compositions, kits and methods are also disclosed.
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
A61P 37/06 - Immunosuppresseurs, p. ex. médicaments pour le traitement du rejet de greffe
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
70.
Production of large-sized quasidystrophins using overlapping AAV vectors
INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) (France)
UNIVERSITE D'EVRY-VAL-D'ESSONNE (France)
Inventeur(s)
Richard, Isabelle
Lostal, William
Abrégé
The present invention concerns a quasidystrophin (QD) having the structure CH1CH2H1R1R2R3H2R8R9 in its N-terminal part and advantageously further comprising the R16 and R17 rod domains, as well as the dual AAV vector system which allows producing it.
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
C12N 5/077 - Cellules mésenchymateuses, p. ex. cellules osseuses, cellules de cartilage, cellules stromales médulaires, cellules adipeuses ou cellules musculaires
A61K 38/00 - Préparations médicinales contenant des peptides
71.
Correction of Beta-Thalassemia Phenotype by Genetically Engineered Hematopoietic Stem Cell
INSERM (Institut National de la Santé et de la Recherche Médicale) (France)
Université d'Evry val d'Essonne (France)
Genethon (France)
Inventeur(s)
Amendola, Mario
Pavani, Giulia
Abrégé
The present invention relates to a genetically modified hematopoietic stem cell (HSC) comprising, in at least one α-globin gene comprised in the genome thereof, at least one transgene encoding a functional β-like globin protein, the said transgene being placed under the control of the endogenous promoter of the said at least one α-globin gene.
A61K 35/28 - Moelle osseuseCellules souches hématopoïétiquesCellules souches mésenchymateuses de toutes origines, p. ex. cellules souches dérivées de tissu adipeux
The present invention concerns a method of treating Duchenne muscular dystrophy (DMD) in a human, comprising systemically administering by intravascular injection a gene therapy product that comprises an adeno-associated viral (AAV) vector of serotype 8, which harbors a nucleic acid sequence encoding a human ΔR4-R23/ΔCT microdystrophin. Advantageously, the gene therapy product is injected up to 1E14 vg/kg.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
75.
BIFUNCTIONAL VECTORS ALLOWING BCL11A SILENCING AND EXPRESSION OF AN ANTI-SICKLING HBB AND USES THEREOF FOR GENE THERAPY OF B-HEMOGLOBINOPATHIES
INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) (France)
UNIVERSITÉ D'EVRY-VAL-D'ESSONNE (France)
ASSISTANCE PUBLIQUE-HÔPITAUX DE PARIS (APHP) (France)
UNIVERSITÉ PARIS CITÉ (France)
FONDATION IMAGINE (France)
GENETHON (France)
Inventeur(s)
Miccio, Annarita
Amendola, Mario
Brusson, Mégane
Cavazzana, Marina
Mavilio, Fulvio
Abrégé
The #β-hemoglobinopathies #β-thalassemia (BT) and sickle cell disease (SCD) are the most frequent genetic disorders worldwide. These diseases are caused by mutations causing reduced or abnormal synthesis of the β-globin chain of the adult hemoglobin (Hb) tetramer. Here, the inventors intend to improve HSC-based gene therapy for β-thalassemia and SCD by developing an innovative, highly infectious LV vector expressing a potent anti-sickling β-globin transgene and a second biological function either increasing fetal γ-globin expression (for β-thalassemia and SCD). More particularly, the inventors have designed a novel lentivirus (LV), which carry two different functions: βAS3 gene addition and gene silencing. This last strategy allows the re-expression of the fetal γ-globin genes (HBG1 and HBG2) and production of the endogenous fetal hemoglobin (HbF). Elevated levels of HbF and HbAS3 (Hb tetramer containing βAS3-globin) will benefit the β-hemoglobinopathy phenotype by increasing the total amount of β-like globin that will: (i) reduce the alpha precipitates and improve the alpha/non alpha ratio in β-thalassemia, and (ii) reduce the sickling in SCD. This combined strategy will improve the β-hemoglobinopathy phenotype at a lower vector copy number (VCN) per cell compared to a LV expressing the βAS3 alone.
A61K 35/28 - Moelle osseuseCellules souches hématopoïétiquesCellules souches mésenchymateuses de toutes origines, p. ex. cellules souches dérivées de tissu adipeux
A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) (France)
Inventeur(s)
Amendola, Mario
Guiraud, Simon
Abrégé
The invention relates to a composition for enhancing utrophin expression in cell by inducing mutations within a target sequence comprising a utrophin repressor binding site using a gene editing enzyme and the use thereof for the treatment of a dystrophinopathy.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
A61K 31/7105 - Acides ribonucléiques naturels, c.-à-d. contenant uniquement des riboses liés à l'adénine, la guanine, la cytosine ou l'uracile et ayant des liaisons 3'-5' phosphodiester
INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) (France)
UNIVERSITE D'EVRY VAL D'ESSONNE (France)
Inventeur(s)
Richard, Isabelle
Dominguez, Natalia
Abrégé
The invention relates to a peptide-modified AAV capsid with improved tropism, in particular increased muscle tropism and/or reduced liver tropism. The invention relates also to the derived recombinant AAV vector particle packaging a gene of interest, and its use in gene therapy, in particular for treating muscle diseases.
C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 9/44 - Hydrolases (3.) agissant sur les composés glycosyliques (3.2) agissant sur les liaisons alpha-glucosidiques-1, 6, p. ex. iso-amylase, pullulanase
A61K 38/00 - Préparations médicinales contenant des peptides
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (France)
UNIVERSITE D'EVRY VAL D'ESSONNE (France)
Inventeur(s)
Buj Bello, Ana
Marinello, Martina
Abrégé
The present invention relates to a recombinant expression cassette comprising a polynucleotide encoding a SMN protein. This cassette can be included in a gene therapy vector and used in a method for the treatment of spinal muscular atrophy (SMA).
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
80.
RECOMBINANT VECTORS SUITABLE FOR THE TREATMENT OF IPEX SYNDROME
INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) (France)
UNIVERSITÉ PARIS CITÉ (France)
ASSISTANCE PUBLIQUE-HÔPITAUX DE PARIS (APHP) (France)
FONDATION IMAGINE (France)
UNIVERSITÉ D'EVRY-VAL-D'ESSONNE (France)
MEDIZINISCHE HOCHSCHULE HANNOVER (Allemagne)
GENETHON (France)
Inventeur(s)
Andre, Isabelle
Six, Emmanuelle
Bellier, Florence
Delville, Marianne
Cavazzana, Marina
Amendola, Mario
Schambach, Axel
Abrégé
IPEX (Immune dysregulation Polyendocrinopathy X linked) syndrome is a primary immunodeficiency caused by mutations in the gene encoding the transcription factor forkhead box P3 (FOXP3), which leads to the loss of function of thymus-derived CD4+CD25+ regulatory T (tTreg) cells. Preclinical and clinical studies suggest that T cell gene therapy approaches designed to selectively restore the repertoire of Treg cells by transfer of wild type FOXP3 gene is a promising potential cure for IPEX. However, there is still a need for a vector that can be used efficiently for the preparation of said Treg cells. The inventors thus compared 6 different lentiviral constructs according to 4 criteria (vector titers, level of transduction of human CD4+ T cells, level of expression of FOXP3 and ΔLNGFR genes, degree of correlation between both expression) and selected one construct comprising a bidirectional PGK-EF1a promoter that showed remarkable efficiency.
C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
C12N 5/0783 - Cellules TCellules NKProgéniteurs de cellules T ou NK
A61K 35/17 - LymphocytesLymphocytes BLymphocytes TCellules tueuses naturellesLymphocytes activés par un interféron ou une cytokine
INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) (France)
UNIVERSITE D'EVRY-VAL-D'ESSONNE (France)
Inventeur(s)
Lostal, William
Richard, Isabelle
Abrégé
The present invention concerns a novel short promoter characterized by a high activity in the skeletal muscles and a low activity in the heart. It then constitutes a valuable candidate especially for driving the expression of transgenes encoding proteins useful for the treatment of muscular dystrophies.
INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) (France)
UNIVERSITE D'EVRY VAL D'ESSONNE (France)
Inventeur(s)
Ronzitti, Giuseppe
Labella, Tiziana
Abrégé
The invention relates to a method of preparation of a recombinant hybrid adeno-associated virus (AAV) capsid protein with improved tropism and to the recombinant hybrid AAV capsid protein obtainable by the method. The invention relates also to the derived expression vector, modified cell, and hybrid capsid AAV vector particle packaging a gene of interest, and its use in tissue-targeted gene therapy for treating various diseases.
INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) (France)
UNIVERSITE D'EVRY-VAL-D'ESSONNE (France)
Inventeur(s)
Richard, Isabelle
Poupiot, Jérôme
Abrégé
The present invention concerns an expression system for systemic administration comprising a sequence encoding gamma-sarcoglycan (SGCG) placed under the control of a promoter allowing an adequate expression of SGCG in the skeletal muscles and in the heart, and its use for the treatment of Limb-Girdle Muscular Dystrophy type C.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61P 9/10 - Médicaments pour le traitement des troubles du système cardiovasculaire des maladies ischémiques ou athéroscléreuses, p. ex. médicaments antiangineux, vasodilatateurs coronariens, médicaments pour le traitement de l'infarctus du myocarde, de la rétinopathie, de l'insuffisance cérébro-vasculaire, de l'artériosclérose rénale
INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) (France)
Inventeur(s)
Richard, Isabelle
Biquand, Ariane
Abrégé
The invention relates to the treatment of dilated cardiomyopathies, in particular genetic dilated cardiomyopathies, using non-expressible inhibitors of the WNT pathway or TGF-β pathway, alone or in combination.
A61K 31/351 - Composés hétérocycliques ayant l'oxygène comme seul hétéro-atome d'un cycle, p. ex. fungichromine ayant des cycles à six chaînons avec un oxygène comme seul hétéro-atome d'un cycle non condensés avec un autre cycle
A61K 31/4439 - Pyridines non condenséesLeurs dérivés hydrogénés contenant d'autres systèmes hétérocycliques contenant un cycle à cinq chaînons avec l'azote comme hétéro-atome du cycle, p. ex. oméprazole
A61K 31/444 - Pyridines non condenséesLeurs dérivés hydrogénés contenant d'autres systèmes hétérocycliques contenant un cycle à six chaînons avec l'azote comme hétéro-atome du cycle, p. ex. amrinone
A61K 31/4725 - Isoquinoléines non condensées, p. ex. papavérine contenant d'autres hétérocycles
A61K 31/4985 - Pyrazines ou pipérazines condensées en ortho ou en péri avec des systèmes hétérocycliques
A61K 31/519 - PyrimidinesPyrimidines hydrogénées, p. ex. triméthoprime condensées en ortho ou en péri avec des hétérocycles
A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
A61P 9/00 - Médicaments pour le traitement des troubles du système cardiovasculaire
INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) (France)
UNIVERSITE D'EVRY VAL D'ESSONNE (France)
Inventeur(s)
Richard, Isabelle
Biquand, Ariane
Abrégé
The invention relates to the treatment of genetic dilated cardiomyopathies using expressible modulators of the Wnt pathway or TGF-β pathway, preferably using gene transfer.
A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
A61P 9/10 - Médicaments pour le traitement des troubles du système cardiovasculaire des maladies ischémiques ou athéroscléreuses, p. ex. médicaments antiangineux, vasodilatateurs coronariens, médicaments pour le traitement de l'infarctus du myocarde, de la rétinopathie, de l'insuffisance cérébro-vasculaire, de l'artériosclérose rénale
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
88.
VECTORS ENCODING A GLUCOSE-6-PHOSPHATASE (G6PASE-A) FOR GENE THERAPY
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (France)
UNIVERSITE EVRY VAL D'ESSONNE (France)
UNIVERSITE CLAUDE BERNARD LYON 1 (France)
Inventeur(s)
Jauze, Louisa
Rajas, Fabienne
Ronzitti, Giuseppe
Abrégé
The invention relates to an adeno-associated virus (AAV) vector comprising a nucleic acid construct for the expression of a glucose-6-phosphatase-a (G6Pase-a) in a cell, the construct comprising a nucleic acid sequence encoding the G6Pase-a, wherein the nucleic acid sequence encoding the G6Pase-a is operably linked to a human alpha-1 antitrypsin (hAAT) promoter, a cell transformed with the vector of the invention, a composition comprising the vector or the cell of the invention, and the use thereof.
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (I.N.S.E.R.M.) (France)
UNIVERSITE D'EVRY VAL D'ESSONNE (UEVE) (France)
UNIVERSITY OF LEICESTER (Royaume‑Uni)
Inventeur(s)
Bertin, Bérangère
Mingozzi, Federico
Piletska, Elena
Piletsky, Sergey
Veron, Philippe
Abrégé
The invention relates to a peptide that specifically binds to an AAV capsid, said peptide comprising an amino acid sequence having at least 80% identity with an amino acid sequence selected from SEQ ID NO : 1, SEQ ID NO : 2, SEQ ID NO : 3, SEQ ID NO : 4, SEQ ID NO : 5 or SEQ ID NO : 6, The invention further relates to a composition comprising one or more peptides of the invention, a kit-of-parts comprising (i) a peptide of the invention or a composition of the invention and (ii) an AAV vector. The invention also relates to Molecularly imprinted polymer (MIP) particle that specifically binds to a peptide of the invention.
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) (France)
UNIVERSITE D'EVRY VAL D'ESSONNE (France)
UNIVERSITY OF LEICESTER (Royaume‑Uni)
Inventeur(s)
Bertin, Bérangère
Mingozzi, Federico
Piletska, Elena
Piletsky, Sergey
Veron, Philippe
Abrégé
The invention relates to a peptide that specifically binds to an AAV capsid, said peptide comprising an amino acid sequence having at least 80% identity with an amino acid sequence selected from SEQ ID NO : 1, SEQ ID NO : 2, SEQ ID NO : 3, SEQ ID NO : 4, SEQ ID NO : 5 or SEQ ID NO : 6. The invention further relates to a composition comprising one or more peptides of the invention, a kit-of-parts comprising (i) a peptide of the invention or a composition of the invention and (ii) an AAV vector. The invention also relates to Molecularly imprinted polymer (MIP) particle that specifically binds to a peptide of the invention.
INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE (INSERM) (France)
SORBONNE UNIVERSITÉ (France)
UNIVERSITE D'EVRY VAL D'ESSONNE (France)
ASSOCIATION INSTITUT DE MYOLOGIE (France)
Inventeur(s)
Mingozzi, Federico
Colella, Pasqualina
Abrégé
The present invention relates to hybrid transcription regulatory elements to drive gene expression, in particular hybrid promoters, designed by the fusion of at least two transcription regulatory elements with different tissue selectivity, such as two promoters driving expression in different tissues in a tissue-selective manner.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12Q 1/68 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions faisant intervenir des acides nucléiques
92.
Synthetic polynucleotides encoding a human FKRP protein
INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) (France)
Inventeur(s)
Richard, Isabelle
Gicquel-Zouida, Evelyne
Lostal, William
Abrégé
The present invention concerns synthetic polynucleotides encoding a human fukutin-related protein (FKRP) wherein the synthetic polynucleotides contain at least a mutation avoiding supplementary transcript(s) generated from frameshift start codon(s). The synthetic polynucleotides are useful, especially for treating a pathology linked to a FKRP deficiency or induced by a defect in α-dystroglycan (α-DG) glycosylation, such as LGMD2I.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) (France)
Inventeur(s)
Buj Bello, Ana
Renaud-Gabardos, Edith
Grimm, Dirk
Weinmann, Jonas
Abrégé
The invention relates to the use of a recombinant porcine adeno-associated virus (AAV) vector comprising a peptide-modified porcine AAV serotype 1 (AAVpo1) capsid in gene therapy of muscle and/or central nervous system (CNS) disorders, in particular neuromuscular diseases such as genetic neuromuscular diseases.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) (France)
GENETHON (France)
UNIVERSITÉ D'EVRY-VAL-D'ESSONNE (France)
Inventeur(s)
Sylvain, Fisson
Abrégé
Despite the eye's immune-privileged status, a secondary loss of vision in some patients treated with AAV led the inventors to question the immunogenicity of AAV vectors after a subretinal injection. The inventors thus characterized anti-transgene and anti-capsid immune responses induced in the periphery after the subretinal AAV injection. Different doses of AAV8 encoding reporter proteins fused with the HY male antigen were injected at day 0 into the subretinal space of adult immunocompetent C57BL/6 female mice. Subretinal AAV injection induced a dose-dependent proinflammatory immune response to the transgene product, correlated with local transgene expression. In order to trigger a subretinal-associated immune inhibition (SRAII) mechanism, some mice were co-injected subretinally at day 0 with AAV and HY peptides. Interestingly, this subretinal co-injection of AAV8 with peptides of the transgene product modulated the anti-transgene T-cell immune response, even at high dose of vector (5.1010 vg). This immunodulation was also confirmed in a pathophysiological murine model of retinal degeneration. The inventors also demonstrated that injection of AAV8 in the subretinal space induces proinflammatory peripheral immune responses to the transgene and the capsid that could be counteracted by co-injection with transgene peptides. Accordingly, the object of the present invention is to provide methods for preventing induction of immune responses to the transgene product and the AAV capsid after ocular gene therapy.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61K 39/39 - Préparations médicinales contenant des antigènes ou des anticorps caractérisées par les additifs immunostimulants, p. ex. par les adjuvants chimiques
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12N 9/44 - Hydrolases (3.) agissant sur les composés glycosyliques (3.2) agissant sur les liaisons alpha-glucosidiques-1, 6, p. ex. iso-amylase, pullulanase
Institut National de la Santé et de la Recherche Médicale (France)
Université d'Evry val d'Essonne (France)
Inventeur(s)
Galy, Anne
Coquin, Youna
Ferrand, Maxime
Abrégé
The invention relates to a pharmaceutical composition for targeting drug delivery including gene delivery to lung tissue, comprising at least a therapeutic drug or gene associated to a syncytin protein, and its use in the prevention and/or treatment of lung diseases, in particular in gene therapy of said diseases using lentiviral vector particles or lentivirus-like particles pseudotyped with syncytin protein.
C12N 7/04 - Inactivation ou atténuationProduction de parties élémentaires de virus
A61P 11/00 - Médicaments pour le traitement des troubles du système respiratoire
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
98.
COMBINATION OF A PROTEASOME INHIBITOR AND A HDAC INHIBITOR AND ITS USE FOR THE TREATMENT OF GENETIC DISEASES LINKED TO A PROTEIN CONFORMATIONAL DISORDER
CENTRE D'ETUDE DES CELLULES SOUCHES (CECS) (France)
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (France)
GENETHON (France)
UNIVERSITE D'EVRY-VAL-D'ESSONNE (France)
Inventeur(s)
Hoch, Lucile
Nissan, Xavier
Richard, Isabelle
Abrégé
The present invention relates to the combination of a proteasome inhibitor and a histone deacetylase (HDAC) inhibitor and its use for the treatment of a genetic disease linked to a conformational disorder of at least one protein, said disorder causing the proteasome degradation of the protein.
BrassicaceaeBrassicaceae family. The invention also relates to a transgenic plant, a hairy root culture and a recombinant viral vector obtainable by the method of the invention.
INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE (France)
UNIVERSITE D'EVRY-VAL-D'ESSONNE (France)
GENETHON (France)
FONDAZIONE ISTITUTO ITALIANO DI TECNOLOGIA (Italie)
FONDAZIONE TELETHON (Italie)
Inventeur(s)
Mingozzi, Federico
Ronzitti, Giuseppe
Contestabile, Andrea
Cancedda, Laura
Abrégé
An RNA interference (RNAi) strategy is provided based on use of artificial microRNA (amiR) to reduce NKCC1 expression. In particular, a method is provided that achieves neuron-specific expression of specific amiR against NKCC1 by using a human Synapsin promoter to drive transgene expression.
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
C12Q 1/68 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions faisant intervenir des acides nucléiques
