The present invention provides inhibitors of IFN-γ signaling reducing the inflammatory insult caused by aberrant DUX4 expression as a novel therapeutic approach for the treatment of FSHD.
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
C07K 16/24 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des cytokines, des lymphokines ou des interférons
An enveloped viral particle producer or packaging cell, wherein the cell is genetically engineered to decrease expression of CD47 on the surface of the cell.
The present invention relates to a CD4+ T cell that produces high levels of IL-10 for use in the treatment and/or prevention of a tumor that expresses CD13, HLA-class I and CD54 and/or for use in inducing Graft versus tumour (GvT). The present invention relates also to a composition comprising said cell and to a method to select a subject to be treated with said cell.
A61K 40/10 - Immunothérapie cellulaire caractérisée par le type de cellules
A61K 40/11 - Lymphocytes T, p. ex. lymphocytes infiltrant les tumeurs [TIL] ou lymphocytes T régulateurs [Treg]Cellules tueuses activées par les lymphokines [LAK]
A61K 40/22 - Immunosuppresseur ou induisant la tolérance
The present disclosure provides methods for generating hematopoietic progenitor cells. In some embodiments, the methods involve an in vitro or ex vivo cell culture model utilizing rentionic acid signaling for producing hematopoietic progenitor cells from pluripotent stem cells.
The invention is directed to means and methods for efficient and safe genetic manipulation of a target cell, wherein a knock-out/knock in strategy is performed via HDR in a target gene that is haploinsufficient (and/or in single copy) in the cell.
An inhibitor of p53 activation for use in haematopoietic stem and/or progenitor cell gene therapy, preferably wherein the inhibitor is an inhibitor of p53 phosphorylation, more preferably an inhibitor of p53 Serine 15 phosphorylation.
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 43/00 - Médicaments pour des utilisations spécifiques, non prévus dans les groupes
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
The present invention relates to combinations, inhibitors, viral vector, methods and uses involving gene therapy and gene editing approaches and immunomodulation within that context.
C07K 16/28 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
C12N 15/87 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation
8.
METHOD FOR PRODUCING T CELL PROGENITORS FROM HUMAN PLURIPOTENT STEM CELLS
FONDATION IMAGINE - INSTITUT DES MALADIES GÉNÉTIQUES (France)
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (France)
UNIVERSITE PARIS CITE (France)
FONDAZIONE TELETHON ETS (Italie)
OSPEDALE SAN RAFFAELE S.R.L. (Italie)
Inventeur(s)
Devi Moirangthem, Ranjita
Andre, Isabelle
Ditadi, Andrea
Cascione, Sara
Abrégé
The present invention relates to a method for generating T cell progenitors from pluripotent stem cells, and to the therapeutic use of the generated T cell progenitors.
An epigenetic silencer factor (ESP), or polynucleotide encoding therefor, for use in the treatment of cancer, wherein the ESF comprises a transcription factor DNA-binding domain operably linked to at least one epigenetic effector domain, wherein the transcription factor is an oncogenic transcription factor or a cancer-associated transcription factor, wherein the cancer is selected from the group consisting of: glioma, gliobastoma, medulloblastoma, astrocytoma, neuroblastomas, ependymoma, meningioma, retinoblastoma, rhabdomyosarcoma, lung cancer, prostate cancer, breast cancer, liver cancer, pancreatic cancer (e.g. human pancreatic ductal adenocarcinoma), bladder cancer, oropharyngeal cancer, kidney cancer, colon cancer (e.g. colon adenocarcinoma), colon-rectal cancer (CRC), or a metastasis of any of the foregoing.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
ALMA MATER STUDIORUM - UNIVERSITÀ DI BOLOGNA (Italie)
Inventeur(s)
Comes Franchini, Mauro
Locatelli, Erica
Tortorella, Silvia
Alfano, Massimo
Venegoni, Chiara
Locatelli, Irene
Abrégé
The invention is directed to a multifunctional system bearing a ligand capable of recognizing tumor cells or tumor-associated or inflammation-associated component and linked to a metal nanoparticle. More specifically, the multifunctional system of the invention comprises a nucleotide aptamer or a variant thereof that binds to the alpha 5 subunit of integrin and a metal nanoparticle, wherein said aptamer optionally comprises at least one modification capable of enhancing nuclease resistance thus providing a molecule with high stability for example in human urine.
C12N 15/115 - Aptamères, c.-à-d. acides nucléiques liant spécifiquement une molécule cible avec une haute affinité sans s'y hybrider
G01N 33/574 - Tests immunologiquesTests faisant intervenir la formation de liaisons biospécifiquesMatériaux à cet effet pour le cancer
G01N 33/543 - Tests immunologiquesTests faisant intervenir la formation de liaisons biospécifiquesMatériaux à cet effet avec un support insoluble pour l'immobilisation de composés immunochimiques
Use of a combination of (a) cyclosporin H (CsH) or a derivative thereof, and (b) a p53 inhibitor and/or an adenoviral protein, or one or more nucleotide sequences encoding therefor, for increasing the efficiency of gene editing of an isolated population of cells when transduced by a viral vector and/or increasing the efficiency of transduction of an isolated population of cells by a viral vector.
A61K 35/28 - Moelle osseuseCellules souches hématopoïétiquesCellules souches mésenchymateuses de toutes origines, p. ex. cellules souches dérivées de tissu adipeux
A method for haematopoietic stem and/or progenitor cell (HSPC) transplantation in a subject in need thereof, comprising the steps: (a) administering one or more HSPC mobiliser to the subject to mobilise the subject's endogenous HSPCs; and (b) administering a population of HSPCs to the subject.
A61K 35/28 - Moelle osseuseCellules souches hématopoïétiquesCellules souches mésenchymateuses de toutes origines, p. ex. cellules souches dérivées de tissu adipeux
A lentiviral vector comprising a nucleotide sequence encoding low density lipoprotein receptor (LDLR) operably linked to a promoter, optionally wherein the nucleotide sequence encoding LDLR and the promoter are in a reverse orientation in the lentiviral vector.
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 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
C07K 16/28 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
The present invention is related to an isolated guide ribonucleic acid (gRNA) including a guide sequence targeting an inhibitory receptor (IR), a TCR α (TRAC) constant region or a β chain (TRBC1/2) constant region target sequence, wherein the guide sequence is selected from the group consisting of SEQ ID NOs: 1-27, 122-126 and combinations thereof.
The present invention relates to an antibody or antigen binding fragment thereof that binds specifically to IGFBP3 and does not displace the binding of IGF-I to IGFBP3. The antibody inhibits or reduces the binding of IGFBP3 to the TMEM219 receptor. The invention also relates to methods for their production, pharmaceutical compositions containing said antibodies, and uses thereof.
C07K 16/22 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des facteurs de croissance
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
A vector for liver and/or splenic phagocyte-specific expression, wherein the vector comprises a transgene operably linked to one or more expression control 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
An adeno-associated viral (AAV) capsid protein, wherein the capsid protein comprises: (a) a peptide comprising or consisting of the amino acid sequence PGVPGRF, or a variant thereof having up to two amino acid substitutions, additions or deletions; or (b) a peptide comprising or consisting of the amino acid sequence NGVRSVG, or a variant thereof having up to two amino acid substitutions, additions or deletions.
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
The present invention provides a chimeric antigen receptor (CAR) comprising an antigen binding domain comprising a chromogranin A (CgA) peptide or a fragment or derivative thereof.
A61K 40/11 - Lymphocytes T, p. ex. lymphocytes infiltrant les tumeurs [TIL] ou lymphocytes T régulateurs [Treg]Cellules tueuses activées par les lymphokines [LAK]
A product comprising two or more artificial transcription repressors (ATRs), or polynucleotides encoding therefor, selected from groups (a), (b), (c) or (d): (a) an ATR comprising a DNA-binding domain operably linked to a KRAB domain or homologue thereof; (b) an ATR comprising a DNA-binding domain operably linked to a DNMT3A, DNMT3B or DNMT1 domain or homologue thereof; (c) an ATR comprising a DNA-binding domain operably linked to a DNMT3L domain or homologue thereof; and (d) an ATR comprising a DNA-binding domain operably linked to a SETDB1 domain or homologue thereof, wherein at least two of the ATRs are selected from different groups (a), (b), (c) or (d).
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 35/12 - Substances provenant de mammifèresCompositions comprenant des tissus ou des cellules non spécifiésCompositions comprenant des cellules souches non embryonnairesCellules génétiquement modifiées
A61K 38/00 - Préparations médicinales contenant des peptides
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 14/47 - 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
The present invention refers to: a) at least one peptide recognizing tumor endothelial cell markers, the peptide being preferably: —an αv-integrin ligand, preferably a ligand of αvβ3, βvβ5, αvβ8, α5β1 and/or αvβ6 integrin, more preferably the peptide includes a sequence having a RGD motif or of a CgA sequence or—a ligand of CD 13 receptor, more preferably the ligand includes a sequence having a NGR motif or functional fragments or derivatives or a biologically active variant thereof and b) saporin or functional fragments, derivatives or a biologically active variant thereof.
C07K 14/435 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
A61K 38/00 - Préparations médicinales contenant des peptides
A61K 40/11 - Lymphocytes T, p. ex. lymphocytes infiltrant les tumeurs [TIL] ou lymphocytes T régulateurs [Treg]Cellules tueuses activées par les lymphokines [LAK]
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
The invention provides a lentiviral vector comprising a construct comprising from 5' to 3': (a) a first nucleotide sequence encoding FoxP3; (b) a second nucleotide sequence encoding a cleavable linker; and (c) a third nucleotide sequence encoding a CD19 CAR.
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 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
It forms an object of the present invention a hollowed scaffold and a method to obtain the same, said hollowed scaffold being a milli-scale vessel-like structure comprising a microenvironment, a lumen, and a vessel wall, said microenvironment and said vessel wall being made, independently the one from the other, of a matrix, wherein in said matrix constituting said microenvironment stromal cells are embedded, and wherein in said matrix constituting said vessel walls endothelial cells are embedded. Additional objects of the present invention are a bioreactor comprising the hollowed scaffold and method to study cells in tissue-like vascularised constructs.
The present invention relates to polynucleotides comprising a nucleotide sequence comprising an expression cassette and a transcription termination site, wherein the nucleotide sequence further comprises: (a) one or more miRNA target sequences having the same orientation as the expression cassette, upstream from the expression cassette and/or downstream from the transcription termination site; (b) one or more miRNA target sequences having the opposite orientation as the expression cassette; and (c) one or more insulator elements.
A vector comprising an astrocyte-specific promoter operably linked to a nucleotide sequence encoding brain-derived neurotrophic factor (BDNF), or a precursor thereof.
The present invention relates to means and methods for the ex vivo or in vitro genetic manipulation of UBA1 gene in somatic cells and to their use in the study and treatment of VEXAS syndrome.
The present invention relates to at least one glycosylation inhibitor for use in combination with CAR cell therapy. Preferably the glycosylation inhibitor improves the therapeutic potential of the CAR cell therapy. The invention also relates to pharmaceutical composition and to population or subpopulation of CAR cell that has been contacted with at least one glycosylation inhibitor.
A61K 31/7004 - Monosaccharides ayant uniquement des atomes de carbone, d'hydrogène et d'oxygène
A61K 31/145 - Amines, p. ex. amantadine ayant des atomes de soufre, p. ex. thiurames (N-C(S)-S-C(S)-N ou N-C(S)-S-S-C(S)-N)Sulfinylamines (-N=SO)Sulfonylamines (-N=SO2)
A61K 31/407 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p. ex. guanéthidine ou rifamycines ayant des cycles à cinq chaînons avec un azote comme seul hétéro-atome d'un cycle, p. ex. sulpiride, succinimide, tolmétine, buflomédil condensés avec des systèmes hétérocycliques, p. ex. kétorolac, physostigmine
A61K 31/437 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p. ex. guanéthidine ou rifamycines ayant des cycles à six chaînons avec un azote comme seul hétéro-atome d'un cycle condensés en ortho ou en péri avec des systèmes hétérocycliques le système hétérocyclique contenant un cycle à cinq chaînons ayant l'azote comme hétéro-atome du cycle, p. ex. indolizine, bêta-carboline
A61K 31/7008 - Composés ayant un groupe amino lié directement à un atome de carbone du radical saccharide, p. ex. D-galactosamine, ranimustine
A61K 31/7072 - Composés ayant des radicaux saccharide et des hétérocycles ayant l'azote comme hétéro-atome d'un cycle, p. ex. nucléosides, nucléotides contenant des cycles à six chaînons avec l'azote comme hétéro-atome d'un cycle contenant des pyrimidines condensées ou non-condensées ayant des groupes oxo liés directement au cycle pyrimidine, p. ex. cytidine, acide cytidylique ayant deux groupes oxo liés directement au cycle pyrimidine, p. ex. uridine, acide uridylique, thymidine, zidovudine
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 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
UNIVERSITA DEGLI STUDI DEL PIEMONTE ORIENTALE AMEDEO AVOGADRO (Italie)
Inventeur(s)
Milani, Michela
Cantore, Alessio
Follenzi, Antonia
Abrégé
in vitro, in vivo ex vivoex vivo, wherein a nucleic acid is transduced by means of the GP64 pseudotyped lentiviral vector particle. Also provided is a method of treating a subject in need of liver regeneration or suffering from a liver disease or haemophilia comprising administration of a GP64 pseudotyped lentiviral vector particle, and the GP64 pseudotyped lentiviral vector particle for use in transducing liver endothelial cells in vivo, such as for liver regeneration or for the treatment of a liver disease or haemophilia. Still further provided is a GP64 pseudotyped lentivirus vector particle comprising a nucleic acid, said nucleic acid comprising the long or short form endogenous human factor VIII (F8) promoter operably linked to a 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
The invention provides targeting RNA, single guide RNA, tracrRNA, crisprRNA and expression vectors for use in CRISPR activation (CRISPRa) methods for the treatment of neurological disorders and diseases, in particular epilepsy and pain. In some preferred embodiments, a combinatorial gene therapy approach is used, wherein expression of multiple endogenous human genes are increased in a subject, in order to achieve a greater rescue of seizures and/or behavioural deficits, and restore physiological brain function.
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 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
The present invention refers to a molecule for use in the treatment and/or prevention of DUX4- r positive leukemia wherein said molecule is selected from the group consisting of: - an inhibitor of GTF2I expression and/or function; - an inhibitor of HDAC expression and/or function; and - an inhibitor of c-Myc expression and/or function.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 31/166 - Amides, p. ex. acides hydroxamiques ayant des cycles aromatiques, p. ex. colchicine, aténolol, progabide ayant l'atome de carbone d'un groupe carboxamide lié directement au cycle aromatique, p. ex. procaïnamide, procarbazine, métoclopramide, labétalol
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
A61P 35/02 - Agents anticancéreux spécifiques pour le traitement de la leucémie
A61K 31/407 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p. ex. guanéthidine ou rifamycines ayant des cycles à cinq chaînons avec un azote comme seul hétéro-atome d'un cycle, p. ex. sulpiride, succinimide, tolmétine, buflomédil condensés avec des systèmes hétérocycliques, p. ex. kétorolac, physostigmine
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 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
C07C 259/06 - Composés contenant des groupes carboxyle, un atome d'oxygène d'un groupe carboxyle étant remplacé par un atome d'azote, cet atome d'azote étant lié de plus à un atome d'oxygène et ne faisant pas partie de groupes nitro ou nitroso sans remplacement de l'autre atome d'oxygène du groupe carboxyle, p. ex. acides hydroxamiques ayant des atomes de carbone de groupes hydroxamique liés à des atomes d'hydrogène ou à des atomes de carbone acycliques
The present invention provides a T-cell receptor (TCR) which binds to an immunogenic peptide when presented by a major histocompatibility complex (MHC).
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 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
The present invention relates to an engineered transposase comprising a transposase operably linked to a polypeptide that binds to a component of heterochromatin. The present invention further relates to an engineered transposome complex comprising an oligonucleotide and an engineered transposase according to the invention. The present invention also relates to methods and uses of the engineered transposase of the invention and engineered transposome of the invention for making a DNA sequence library or libraries and for DNA sequencing.
The present invention relates to an isolated polynucleotide comprising from 5′ to 3′: a first homology region, a nucleotide sequence encoding a RAG1 polypeptide or a RAG1 polypeptide fragment, and a second homology region for use in treating a RAG-deficient immunodeficiency.
The present invention relates to a method for genetic manipulation of immune cells or progenitors thereof, by lipid nanoparticles delivery of a gene modifying agent to cells.
Use of one or more inhibitor(s) of senescence for increasing the survival and/or engraftment of haematopoietic cells, haematopoietic stem cells, haematopoietic progenitor cells and/or T cells in gene therapy.
A61K 35/28 - Moelle osseuseCellules souches hématopoïétiquesCellules souches mésenchymateuses de toutes origines, p. ex. cellules souches dérivées de tissu adipeux
A vector for central nervous system (CNS)-specific expression, wherein the vector comprises a transgene encoding interleukin 10 (IL-10), and wherein the transgene is operably linked to one or more expression control sequence.
The present invention concerns a system for classifying a cataract. The system comprises a head positioning module (100) suitable for receiving a head of a patient and positioning it, a source module (201) capable of emitting electromagnetic radiation, with a frequency between 400nm and 490nm, and preferably between 450nm and 470nm and more preferably equal to 460nm, to focus it on a lens of the patient and a detector module (202) suitable for acquiring the image of the lens illuminated by electromagnetic radiation. A control unit (204) configured to control the source module (201) and the detector module (202), and to process the lens image to determine the grade of cataract based on pixel values of the image. A computerized method for classifying a cataract is also described.
A61B 3/10 - Appareils pour l'examen optique des yeuxAppareils pour l'examen clinique des yeux du type à mesure objective, c.-à-d. instruments pour l'examen des yeux indépendamment des perceptions ou des réactions du patient
A61B 3/117 - Appareils pour l'examen optique des yeuxAppareils pour l'examen clinique des yeux du type à mesure objective, c.-à-d. instruments pour l'examen des yeux indépendamment des perceptions ou des réactions du patient pour examiner la chambre antérieure ou l'angle de la chambre antérieure, p. ex. gonioscopes
A product comprising: (a) a vector for liver and/or splenic phagocyte-specific expression, wherein the vector comprises a transgene operably linked to one or more expression control sequence; and (b) an immune checkpoint inhibitor or a Tr1 cell inhibitor.
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 38/00 - Préparations médicinales contenant des peptides
An enveloped viral particle producer or packaging cell, wherein the cell is modified to decrease expression of low density lipoprotein receptor (LDLR) on the surface of the cell.
Use of cyclosporin H (CsH) or a derivative thereof for increasing the efficiency of transduction of an isolated population of cells by a viral vector and/or increasing the efficiency of gene editing of an isolated population of cells when transduced by a viral vector.
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 7/64 - Peptides cycliques ne comportant que des liaisons peptidiques normales
An enveloped viral particle producer or packaging cell, wherein the cell is genetically engineered to decrease expression of MHC-I on the surface of the cell.
A polynucleotide comprising a nucleotide sequence encoding methyl-CpG binding-protein 2 (MeCP2) and: (a) at least one miR-124 target sequence, and/or at least one miR-31 target sequence, and/or at least one miR-338-3p target sequence; and/or (b) a nucleotide sequence encoding an inhibitor of MeCP2 expression, optionally a nucleotide sequence encoding an shRNA that has at least 90% sequence identity to SEQ ID NO: 15, 30 or 31.
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 38/00 - Préparations médicinales contenant des peptides
A product comprising: (a) a hematopoietic stem cell (HSC), a hematopoietic progenitor cell (HPC), a myeloid/monocyte-committed progenitor cell, a macrophage or a monocyte comprising a vector, wherein the vector comprises at least one miRNA target sequence operably linked to a transgene, wherein the miRNA target sequence prevents or reduces expression of the transgene in the HSC, HPC and/or myeloid/monocyte-committed progenitor cell, wherein the transgene encodes an orthogonal cytokine; and (b) a T cell or NK cell, wherein the T cell or NK cell expresses an orthogonal receptor.
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
A61K 35/28 - Moelle osseuseCellules souches hématopoïétiquesCellules souches mésenchymateuses de toutes origines, p. ex. cellules souches dérivées de tissu adipeux
A61K 35/12 - Substances provenant de mammifèresCompositions comprenant des tissus ou des cellules non spécifiésCompositions comprenant des cellules souches non embryonnairesCellules génétiquement modifiées
C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
C07K 14/715 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire pour des cytokinesRécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire pour des lymphokinesRécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire pour des interférons
The present invention relates to novel antagonists of the Liver X Receptors (LXRs) of formula (I) and (II) which can be used alone or in combination with other anti cancer therapies, such as the immune checkpoint blockers or cell adoptive cell therapy, preferably T cell adoptive cell therapy, to treat different cancers, including melanoma, Hodgkin lymphoma, renal, lung, bladder and head and neck cancers.
C07J 9/00 - Stéroïdes normaux contenant du carbone, de l'hydrogène, un halogène ou de l'oxygène, substitués en position 17bèta par une chaîne de plus de deux atomes de carbone, p. ex. cholane, cholestane, coprostane
C07J 31/00 - Stéroïdes normaux contenant un ou plusieurs atomes de soufre n'appartenant pas à un hétérocycle
A61P 5/44 - GlucocorticoïdesMédicaments augmentant ou potentialisant l'activité des glucocorticoïdes
A61K 31/575 - Composés contenant des systèmes cycliques du cyclopenta[a]hydrophénanthrèneLeurs dérivés, p. ex. stéroïdes substitués en position 17 bêta par une chaîne d'au moins trois atomes de carbone, p. ex. cholane, cholestane, ergostérol, sitostérol
C07J 53/00 - Stéroïdes ayant le squelette du cyclopenta[a]hydrophénanthrène modifié par condensation avec des cycles carbocycliques ou par formation d'un cycle additionnel par une liaison directe entre deux atomes de carbone cycliques
47.
COMPOSITIONS AND METHODS FOR TREATING DISEASES AND DISORDERS OF THE CENTRAL NERVOUS SYSTEM
The present invention provides compositions and methods for the treatment or prevention of a neurological disease or disorder of the central nervous system (e.g., a storage disorder, lysosomal storage disorder, neurodegenerative disease, etc.) by reconstitution of brain myeloid cell and microglia upon transplantation of hematopoietic cells enriched in microglia reconstitution potential. The invention also provides compositions and methods for ablating and reconstituting microglia.
A61K 35/12 - Substances provenant de mammifèresCompositions comprenant des tissus ou des cellules non spécifiésCompositions comprenant des cellules souches non embryonnairesCellules génétiquement modifiées
A01K 67/0271 - Vertébrés chimériques, p. ex. comprenant des cellules exogènes
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
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
The present invention provides a T-cell receptor (TCR) which binds to a Human Telomerase Reverse Transcriptase (hTERT) peptide or a Survivin peptide when presented by a major histocompatibility complex (MHC).
An image capturing method (100) using an image capturing apparatus (1) that comprises an imaging device (2) having an image catcher (3) for generating image data, the method (100) comprising displaying (S101), using an image reproducing device (4), images based on the image data, generating (S102) attribute data from a plurality of biological signals measured by a plurality of biological sensors (6), the attribute data being at least an information extracted by one or more biological signals through a computational processing and being indicative of attributes of a user at a time that the user is viewing the images displayed by the image reproducing device (4), generating (S103) significance data based on the attribute data, and associating the attribute data from which the significance data is generated with each displayed image, wherein the significance data are generated by comparing the attribute data with predetermined significance threshold values in order to identify a significance state for each attribute data, controlling (S104) photographic parameters of the image capturing apparatus (1) based on the attribute data, wherein controlling the photographic para-meters comprises at least a variation of said photographic parameters as a function of a variation of at least one biological signal, analysing (S105) the significance data to determine if the displayed images should be stored, wherein analysing the significance data comprises generating at least a significance level from the significance data and comparing said significance level with a triggering threshold level, and storing (S106) the image data of the displayed images together with the attribute data to a storage device (7) based on the analysed significance data.
H04N 23/611 - Commande des caméras ou des modules de caméras en fonction des objets reconnus les objets reconnus comprenant des parties du corps humain
H04N 23/60 - Commande des caméras ou des modules de caméras
H04N 23/63 - Commande des caméras ou des modules de caméras en utilisant des viseurs électroniques
A combination of: (a) at least one deoxyribonucleoside (dN) or a derivative thereof and cyclosporin H (CsH) or a derivative thereof: or (b) at least one pyrimidine precursor and cyclosporin H (CsH) or a derivative thereof.
A61K 31/513 - PyrimidinesPyrimidines hydrogénées, p. ex. triméthoprime ayant des groupes oxo liés directement à l'hétérocycle, p. ex. cytosine
A61K 31/7072 - Composés ayant des radicaux saccharide et des hétérocycles ayant l'azote comme hétéro-atome d'un cycle, p. ex. nucléosides, nucléotides contenant des cycles à six chaînons avec l'azote comme hétéro-atome d'un cycle contenant des pyrimidines condensées ou non-condensées ayant des groupes oxo liés directement au cycle pyrimidine, p. ex. cytidine, acide cytidylique ayant deux groupes oxo liés directement au cycle pyrimidine, p. ex. uridine, acide uridylique, thymidine, zidovudine
A61K 35/28 - Moelle osseuseCellules souches hématopoïétiquesCellules souches mésenchymateuses de toutes origines, p. ex. cellules souches dérivées de tissu adipeux
The present invention refers to a peptide or variants thereof wherein the peptide essentially consists of: - an amino acid sequence comprised in the sequence from aa. 1 to aa. 288 of matrin 3 (MATR3) (SEQ ID NO:1), or - a variant of an amino acid sequence comprised in the sequence from aa. 1 to aa. 288 of matrin 3 (MATR3), with the proviso that the peptide does not consist of the amino acid sequence from aa.1 to aa. 287 of matrin 3 (SEQ ID NO:17), and preferably wherein amino acid sequence is comprised in the sequence from aa. 1 to aa. 287 or from aa 2 to aa. 288 (SEQ ID NO:18) or from aa. 2 to aa. 287 (SEQ ID NO:19)
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 38/00 - Préparations médicinales contenant des peptides
A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
A61P 35/02 - Agents anticancéreux spécifiques pour le traitement de la leucémie
C12N 15/70 - Vecteurs ou systèmes d'expression spécialement adaptés à E. coli
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
Method (100) for analyzing a user's reaction to at least one stimulus, wherein the method (100) comprises: providing (S101) at least one stimulus (ST) to the user selected from a plurality of stimuli according to an input information (I- Inf o ) and at least one stimulation parameter (SP) from a plurality of stimulation parameters through one or more sensory stimulators (2); measuring (S102) a biological response following the providing of said stimulus (ST) and acquiring at least one biosignal through one or more biometric sensors (3), wherein the biosignal indicates a neurophysiological reaction to the stimulus (ST) and comprises neurophysiological reaction data (RD); associating (S103) the neurophysiological reaction data (RD) with the corresponding stimulus (ST) through at least one processor (4) to generate at least one piece of sensory metadata (MD) indicative of the user's response to the corresponding stimulus (ST); archiving (S104) the sensory metadata (MD) within a memory support (5) to obtain a plurality of sensory metadata (MD) associated with the user; processing (S105) each piece of sensory metadata (MD) stored in the memory support (5) through the processor (4) according to the input information (I-Info); generating (S106) an output information (O-Info) following the processing of each piece of sensory metadata (MD), the output information (O-Info) comprising a reaction index (RI) to the stimulus as a function of the input information ( I-Info), and modifying (S107) at least one stimulation parameter (SP) according to the reaction index (RI).
A polynucleotide comprising a nucleotide sequence encoding an engineered sacsin, wherein the engineered sacsin comprises a UbL domain or variant thereof, an SRR domain or variant thereof, a DnaJ domain or variant thereof and an HERN domain or variant thereof, and wherein the nucleotide sequence encoding the engineered sacsin is less than or equal to about 4000 bp in length.
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
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 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
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 chimeric antigen receptors (CARs) that target Cadherin-17 (CDH17) and cells comprising said CARs. Methods and uses involving the CARs of the invention are also provided.
C07K 16/28 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants 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
The present invention is directed to a method for ex-vivo-engineering of cells, in particular stem cells or T cells, preferably hematopoietic stem and/or progenitor cells, mesenchymal stem cells, or T cells comprising a step of culturing the cells on a three-dimensional scaffold. The method of the invention is capable of improving the efficiency of genetic modification of cells and the functionality of the engineered cells.
A61K 35/28 - Moelle osseuseCellules souches hématopoïétiquesCellules souches mésenchymateuses de toutes origines, p. ex. cellules souches dérivées de tissu adipeux
A photoacoustic agent chosen among the group consisting of a metal-based nanoparticle made of gold, silver, or hybrid gold/silver, an organic photoacoustic dye, cyanine dyes, phthalein and xanthene dyes, squaraine and croconaine dyes, tetrapyrrole, BODIPY dyes, curcumin dyes, and IRDye800 linked to a ligand of the integrin family receptors, preferably a peptide containing an integrin binding motif, and antibody or part of an antibody, a peptidomimetic or an aptamer, via a crosslinker selected from the group consisting of a crosslinker bearing amino and sulfhydryl reactive groups, bearing amino and azide/alkyne reactive groups, bearing lipoamide/lipoic acid (LA) or sulfhydryl or disulphide containing compounds.
A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
C07K 7/56 - Peptides cycliques contenant au moins une liaison peptidique anormale comportant au moins une liaison peptidique anormale dans le cycle cyclisés autrement que par l'acide diamino-2,4 butanoïque
C07K 7/64 - Peptides cycliques ne comportant que des liaisons peptidiques normales
59.
BIOMATERIAL COATED NANOSTRUCTURES FOR PHOTOACOUSTIC IMAGING AND PHOTOTHERMAL THERAPY OF TUMOR LESIONS
ALMA MATER STUDIORUM - UNIVERSITA' DI BOLOGNA (Italie)
Inventeur(s)
Comes Franchini, Mauro
Maturi, Mirko
Locatelli, Erica
Alfano, Massimo
Alchera, Elisa
Locatelli, Irene
Curnis, Flavio
Corti, Angelo
Abrégé
Metal based nanoparticles coated with a polymer functionalized with thiol groups and 5 -NH groups, said polymer preferably being selected among thiolated chitosan, thiolated and aminated alginic acid, thiolated and aminated hyaluronic acid, or a protein preferably selected from the group consisting of albumin and gelatin, or a synthetic thiolated and aminated polymer, preferably α-thio-ω-amino polyethylene glycols; wherein said groups are linked to a ligand of the integrin family receptors, 10 preferably a peptide containing an integrin binding motif, and antibody or part of an antibody, a peptidomimetic or an aptamer, via a heterobifunctional crosslinker, the crosslinker bearing functional groups able to bind to amino groups, preferably selected from the group consisting of N-hydroxysuccinimidyl ester group (NHS ester), an isocyanate group (-NCO), an isothiocyanate group (-NCS), a Sulfo-N-15 hydroxysuccinimidyl ester group (sulfo-NHS ester), or a carboxylic acid group which is connected by activation with carbodiimide coupling agents; and/or a functional group able to bind thiol groups, preferably selected from the group consisting of: a maleimide group, a terminal vinyl group or a terminal alkyne group; and/or functional groups able to bind alkynes such as azides; and/or functional groups able 20 to bind azides, such as alkynes.
A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
A61K 41/00 - Préparations médicinales obtenues par traitement de substances par énergie ondulatoire ou par rayonnement corpusculaire
A61K 49/22 - Préparations pour échographiePréparations pour imagerie par ultrasons
60.
TESTING METHOD FOR STAINING IN DENTAL RESTORATIVE MATERIALS AND RELATED TESTING SYSTEM
A testing method for staining in dental restorative materials, said testing method comprising: providing a sample of dental restorative material in an initial state; subjecting said sample to at least one cyclical immersion into at least one first staining liquid and into at least one second staining liquid, controlling a periodicity and an overall duration of said cyclical immersion. A testing system for staining in dental restorative materials.
G01N 3/56 - Recherche de la résistance à l'usure ou à l'abrasion
G01N 35/00 - Analyse automatique non limitée à des procédés ou à des matériaux spécifiés dans un seul des groupes Manipulation de matériaux à cet effet
An epigenetic silencer factor (ESF) comprising a transcription factor DNA-binding domain operably linked to at least one epigenetic effector domain, wherein the transcription factor is an oncogenic transcription factor or a cancer-associated transcription factor.
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
The present invention relates to deglycosylating enzymes and modified versions thereof. The invention also provides deglycosylating enzymes alongside cell-binding molecules, such as CARs, for improving the therapeutic activity of CAR-containing cells. Methods and uses involving the deglycosylating enzymes of the invention are also provided.
C12N 9/80 - Hydrolases (3.) agissant sur les liaisons carbone-azote autres que les liaisons peptidiques (3.5) agissant sur les liaisons amides des amides aliphatiques
A61K 35/17 - LymphocytesLymphocytes BLymphocytes TCellules tueuses naturellesLymphocytes activés par un interféron ou une cytokine
The invention relates to means and methods for gene therapy of lysosomal storage disorders (LSDs), preferably a LSD with skeletal involvement, based on an ex vivo gene therapy approach comprising transduction of autologous hematopoietic stem and progenitor cells (HSPCs) with viral vectors for expressing enzymes that are deficient in the disorders. The final formulation is a suspension of transduced cells in culture medium for the administration to patients affected by the LSDs, preferably preceded by a conditioning regimen.
Shade guide device (60) for dental restoration, comprising a core (31) of opaque composite corresponding to a dentine of a dental anatomy, the core (31) being moulded and shaped with portions with differentiated thickness (A, B, C) imitating mamelons of anterior tooth, and further comprising a buccal layer (40) of translucent enamel applied on the core (31) and forming an additional thickness on at least one buccal side of the shade guide device (60) at the portions with differentiated thickness (A, B, C). A shade guide kit. A manufacturing method of a shade guide device.
An epigenetic silencer factor (ESP), or polynucleotide encoding therefor, for use in the treatment of cancer, wherein the ESF comprises a transcription factor DNA-binding domain operably linked to at least one epigenetic effector domain, wherein the transcription factor is an oncogenic transcription factor or a cancer-associated transcription factor, wherein the cancer is selected from the group consisting of: glioma, gliobastoma, medulloblastoma, astrocytoma, neuroblastomas, ependymoma, meningioma, retinoblastoma, rhabdomyosarcoma, lung cancer, prostate cancer, breast cancer, liver cancer, pancreatic cancer (e.g. human pancreatic ductal adenocarcinoma), bladder cancer, oropharyngeal cancer, kidney cancer, colon cancer (e.g. colon adenocarcinoma), colon-rectal cancer (CRC), or a metastasis of any of the foregoing.
C07K 14/34 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de bactéries provenant de Corynebacterium (G)
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 15/67 - Méthodes générales pour favoriser l'expression
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
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
A polynucleotide comprising at least one miR-124 target sequence, and/or at least one miR- 338-3p target sequence, and/or at least one miR-31 target sequence, wherein the miRNA target sequences are operably linked to a transgene.
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
An agent that increases the number ofKupffer cells in a subject, or a nucleotide sequence encoding therefor, for use in a method of therapy by increasing liver immune response.
The present invention refers to An in vitro method of isolating hemogenic endothelial cells (HECs) or enriched populations of HECs, comprising isolating CD32+ cells from a population of cells derived from pluripotent stem cells.
Use of cyclosporin H (CsH) or a derivative thereof for: (a) reducing or preventing T cell exhaustion and/or loss of T cell effector functions; and/or (b) increasing T cell engraftment and/or persistence.
The present invention relates to an isolated polynucleotide comprising from 5′ to 3′: a first homology region, a splice acceptor sequence, a nucleotide sequence encoding a RAG1 polypeptide, and a second homology region for use in treating a RAG-deficient immunodeficiency.
Disclosed herein are methods and compositions for inactivating TCR genes, using zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain in conditions able to preserve cell viability. Polynucleotides encoding ZFNs, vectors comprising polynucleotides encoding ZFNs and cells comprising polynucleotides encoding ZFNs and/or cells comprising ZFNs are also provided. Disclosed herein are also methods and compositions for expressing a functional exogenous TCR in the absence of endogenous TCR expression in T lymphocytes, including lymphocytes with a central memory phenotype. Polynucleotides encoding exogenous TCR, vectors comprising polynucleotides encoding exogenous TCR and cells comprising polynucleotides encoding exogenous TCR and/or cells comprising exogenous TCR are also provided.
A61K 35/28 - Moelle osseuseCellules souches hématopoïétiquesCellules souches mésenchymateuses de toutes origines, p. ex. cellules souches dérivées de tissu adipeux
An adeno-associated viral (AAV) capsid protein, wherein the capsid protein comprises: (a) a peptide comprising or consisting of the amino acid sequence PGVPGRF, or a variant thereof having up to two amino acid substitutions, additions or deletions; or (b) a peptide comprising or consisting of the amino acid sequence NGVRSVG, or a variant thereof having up to two amino acid substitutions, additions or deletions.
A method for haematopoietic stem and/or progenitor cell (HSPC) transplantation in a subject in need thereof, comprising the steps: (a) administering one or more HSPC mobiliser to the subject to mobilise the subject's endogenous HSPCs; and (b) administering a population of HSPCs to the subject.
A61K 35/28 - Moelle osseuseCellules souches hématopoïétiquesCellules souches mésenchymateuses de toutes origines, p. ex. cellules souches dérivées de tissu adipeux
Use of a combination of (a) cyclosporin H (CsH) or a derivative thereof, and (b) a p53 inhibitor and/or an adenoviral protein, or one or more nucleotide sequences encoding therefor, for increasing the efficiency of gene editing of an isolated population of cells when transduced by a viral vector and/or increasing the efficiency of transduction of an isolated population of cells by a viral vector.
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
A lentiviral vector comprising a nucleotide sequence encoding low density lipoprotein receptor (LDLR) operably linked to a promoter, optionally wherein the nucleotide sequence encoding LDLR and the promoter are in a reverse orientation in the lentiviral vector.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A product comprising two or more artificial transcription repressors (ATRs), or polynucleotides encoding therefor, selected from groups (a), (b), (c) or (d): (a) an ATR comprising a DNA-binding domain operably linked to a KRAB domain or homologue thereof; (b) an ATR comprising a DNA-binding domain operably linked to a DNMT3A, DNMT3B or DNMT1 domain or homologue thereof; (c) an ATR comprising a DNA-binding domain operably linked to a DNMT3L domain or homologue thereof; and (d) an ATR comprising a DNA-binding domain operably linked to a SETDB1 domain or homologue thereof, wherein at least two of the ATRs are selected from different groups (a), (b), (c) or (d).
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
A61K 35/12 - Substances provenant de mammifèresCompositions comprenant des tissus ou des cellules non spécifiésCompositions comprenant des cellules souches non embryonnairesCellules génétiquement modifiées
A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
A cartridge is adapted to house at least one biological sample therein, where the cartridge contains at least two overlapping layers, and the layers include at least one layer of highly hydrophobic, inert, and biocompatible material, with contact angle Θc≥90°, hydrophobic layer, and optionally, at least one layer of double-sided adhesive material, where in the absence of the at least one layer of double-sided adhesive material, the overlapping layers are connected together by chemical and/or physical bonding, where each of the overlapping layers has at least one inner hole that is pervious when the layers overlap one another, and the at least one inner hole is closed by the at least one biological sample, where loaded in the cartridge.
The present invention refers to: a) at least one peptide recognizing tumor endothelial cell markers, said peptide being preferably: - an αv-integrin ligand, preferably a ligand of αvβ3, αvβ5, αvβ8, α5β1 and/or αvβ6 integrin, more preferably said peptide comprising or consisting of a sequence comprising a RGD motif or of a CgA sequence or - a ligand of CD 13 receptor, more preferably said ligand comprising or consisting of a sequence comprising a NGR motif or functional fragments or derivatives or a biologically active variant thereof and b) saporin or functional fragments, derivatives or a biologically active variant thereof.
C12N 15/62 - Séquences d'ADN codant pour des protéines de fusion
C07K 14/435 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
A61K 38/16 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés
A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
C07K 7/08 - Peptides linéaires ne contenant que des liaisons peptidiques normales ayant de 12 à 20 amino-acides
C07K 14/415 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de végétaux
79.
NON-INVASIVE VENTILATION SYSTEM FOR THE PRE-HOSPITAL MANAGEMENT OF ACUTE RESPIRATORY FAILURE
A stand-alone continuous positive airways pressure, CPAP, apparatus having a face-mask and a connected electro-mechanical device to supply air to the face-mask is disclosed. The electro-mechanical device includes a pneumatic channel for flowing air to be delivered to the face mask and a control unit for managing the air pressure of the air inside the pneumatic channel. The CPAP apparatus includes a turbine fan, located in the electro-mechanical device housing, connected to the control unit for pressurizing atmospheric air. The pneumatic channel includes an inlet portion located upstream of the turbine fan to receive atmospheric air, and an outlet portion located downstream of the turbine fan to deliver the pressurized air to the face-mask through an outlet opening. The pneumatic channel also longitudinally extends from the inlet portion to the outlet portion.
The invention provides targeting RNA, single guide RNA, tracrRNA, crisprRNA and expression vectors for use in CRISPR activation (CRISPRa) methods for the treatment of neurological disorders and diseases, in particular epilepsy and pain. In some preferred embodiments, a combinatorial gene therapy approach is used, wherein expression of multiple endogenous human genes are increased in a subject, in order to achieve a greater rescue of seizures and/or behavioural deficits, and restore physiological brain function.
The present invention relates to a conjugate comprising a first peptide of sequence CNGRCG (SEQ ID NO: 1) linked to the N-terminus of a protein and a compound X linked to the N-terminus of said peptide and to related medical uses.
The present invention relates to an inhibitor of asparagine synthase for use for the treatment of a disorder characterized by renal and/or liver cyst formation and relative pharmaceutical composition.
A polynucleotide comprising a nucleotide sequence encoding an epidermal growth factor receptor (EGFR) extracellular epitope operably linked to: (a) a NGFR or GMS SFR alpha signal peptide; (b) a EGFR or NGFR transmembrane domain; and/or (c) a NGFR or EGFR cytosplasmic tail.
C07K 14/71 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire pour des facteurs de croissanceRécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire pour des régulateurs de croissance
C07K 16/28 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des récepteurs, des antigènes de surface cellulaire ou des déterminants de surface cellulaire
A61P 37/06 - Immunosuppresseurs, p. ex. médicaments pour le traitement du rejet de greffe
The present invention refers to a method to produce a T cell with advantageous properties. The invention also refers to a T cell or an engineered T cell produced by the method and its use in therapy.
A method for analysing insertion sites of an exogenous nucleotide sequence in a subject's genome, wherein the method comprises: (a) providing a sample from the subject comprising cell-free double-stranded DNA polynucleotides; (b) blunting the ends of the polynucleotides; (c) ligating an oligonucleotide to both ends of the polynucleotides; (d) amplifying polynucleotides comprising an insertion site; and (e) sequencing the product of step (d).
Istituto Nazionale Genetica Molecolare—INGM (Italie)
Inventeur(s)
Di Fabio, Romano
Summa, Vincenzo
Bencheva, Leda Ivanova
De Francesco, Raffaele
Donnici, Lorena
Guidotti, Luca
Iannacone, Matteo
Prandi, Adolfo
De Matteo, Marilenia
Randazzo, Pietro
Abrégé
The present invention relates to compounds that are inhibitors of hepatitis B virus (HBV). Compounds of this invention are useful alone or in combination with other agents for treating, ameliorating, preventing or curing HBV infection and related conditions. The present invention also relates to pharmaceutical compositions containing the compounds.
The present invention refers to an inhibitor of substance P and/or of its receptor for use in the treatment and/or prevention of stem cell deficiency. Preferably the inhibitor is an NK1 antagonist. Preferably the stem cell deficiency is a corneal epithelial stem cell deficiency. The invention also refers to pharmaceutical compositions containing the inhibitor for use in the treatment and/or prevention of stem cell deficiency.
The present invention provides neutralizing monoclonal antibodies targeting the spike protein of Sarbecoviruses, such as Severe Acute Respiratory Syndrome-Coronavirus-2 (SARS-CoV-1 or SARS-CoV-2 like COVID-19). The monoclonal antibodies of the invention can inhibit or neutralize SARS-CoV-1 or SARS-CoV-2 activity and advantageously be used for treating, preventing or diagnosing COVID-19 infection in humans due to their significant cross-reactivity among SARS-CoV-2 variants.
Use of one or more inhibitor(s) of senescence for increasing the survival and/or engraftment of haematopoietic cells, haematopoietic stem cells, haematopoietic progenitor cells and/or T cells in gene therapy.
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
C12N 5/10 - Cellules modifiées par l'introduction de matériel génétique étranger, p. ex. cellules transformées par des virus
C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
The present invention relates to an isolated polynucleotide comprising from 5' to 3': a first homology region, a nucleotide sequence encoding a RAG1 polypeptide or a RAG1 polypeptide fragment, and a second homology region for use in treating a RAG-deficient immunodeficiency.
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
The present invention relates to methods for the screening, diagnosis and/or prognosis of renal cell carcinoma. The methods of the invention are based on the determination of expression profiles of sets of miRNAs representative for renal cell carcinoma optionally associated with genotype analysis, in particular of uromodulin SNP variants. The invention also refers to a kit to perform such methods.
C12Q 1/6886 - Produits d’acides nucléiques utilisés dans l’analyse d’acides nucléiques, p. ex. amorces ou sondes pour les maladies provoquées par des altérations du matériel génétique pour le cancer
The present invention refers to chromogranin A-derived peptides that are potent dual ligands for integrins αvβ6 and avβ8, their therapeutic and diagnostic uses and relative compositions.
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 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
An image capturing method (100) using an image capturing apparatus (1) that comprises an imaging device (2) having an image catcher (3) for generating image data, the method (100) comprising displaying (S101), using an image reproducing device (4), images based on the image data, generating (S102) attribute data from a plurality of biological signals measured by a plurality of biological sensors (6), the attribute data being at least an information extracted by one or more biological signals through a computational processing and being indicative of attributes of a user at a time that the user is viewing the images displayed by the image reproducing device (4), generating (S103) significance data based on the attribute data, and associating the attribute data from which the significance data is generated with each displayed image, wherein the significance data are generated by comparing the attribute data with predetermined significance threshold values in order to identify a significance state for each attribute data, controlling (S104) photographic parameters of the image capturing apparatus (1) based on the attribute data, wherein controlling the photographic parameters comprises at least a variation of said photographic parameters as a function of a variation of at least one biological signal, analysing (S105) the significance data to determine if the displayed images should be stored, wherein analysing the significance data comprises generating at least a significance level from the significance data and comparing said significance level with a triggering threshold level, and storing (S106) the image data of the displayed images together with the attribute data to a storage device (7) based on the analysed significance data.
A combination of: (a) at least one deoxyribonucleoside (dN) or a derivative thereof and cyclosporin H (CsH) or a derivative thereof; or (b) at least one pyrimidine precursor and cyclosporin H (CsH) or a derivative thereof.
A gene vector for use in gene therapy comprising at least one miRNA sequence target operably linked to a nucleotide sequence having a corresponding miRNA in a hematopoietic progenitor cell (HSPC) or hematopoietic stem cell (HSC) which prevents or reduces expression of the nucleotide sequence in a HSPC or HSC but not in a differentiated cell.
THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY (USA)
OSPEDALE SAN RAFFAELE S.R.L. (Italie)
FONDAZIONE TELETHON (Italie)
Inventeur(s)
Bacchetta, Rosa
Roncarolo, Maria Grazia
Sato, Yohei
Naldini, Luigi
Passerini, Laura
Abrégé
Engineered Treg-like cells, CD4LVFOXP3 T cells, and their use in cellular therapy to promote immune tolerance are disclosed. In particular, CD4LVFOXP3 T cells are produced by transduction of CD4+ T cells with a lentiviral vector expressing FOXP3 under the control of a constitutive promoter. Transduced cells express FOXP3 at high and persistent levels and acquire immune suppressive characteristics resembling naturally occurring Treg cells.
An epigenetic silencer factor (ESF) comprising a transcription factor DNA-binding domain operably linked to at least one epigenetic effector domain, wherein the transcription factor is an oncogenic transcription factor or a cancer-associated transcription factor.
C12N 15/62 - Séquences d'ADN codant pour des protéines de fusion
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
An interleukin which binds to IL-2 receptor (IL-2R), or a nucleotide sequence encoding therefor, wherein the interleukin or nucleotide sequence is adapted to be targeted to the liver.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 35/17 - LymphocytesLymphocytes BLymphocytes TCellules tueuses naturellesLymphocytes activés par un interféron ou une cytokine
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 31/20 - Antiviraux pour le traitement des virus ADN
The present invention relates to an engineered transposase comprising a transposase operably linked to a polypeptide that binds to a component of heterochromatin. The present invention further relates to an engineered transposome complex comprising an oligonucleotide and an engineered transposase according to the invention. The present invention also relates to methods and uses of the engineered transposase of the invention and engineered transposome of the invention for making a DNA sequence library or libraries and for DNA sequencing.
C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
C40B 20/04 - Identification des éléments d'une bibliothèque au moyen d'une étiquette, d'un marqueur ou d'un autre identificateur lisible ou détectable, p. ex. procédés de décodage
C40B 30/04 - Procédés de criblage des bibliothèques en mesurant l'aptitude spécifique à se lier à une molécule cible, p. ex. liaison anticorps-antigène, liaison récepteur-ligand
The present invention is related to an isolated guide ribonucleic acid (gRNA) comprising a guide sequence targeting an inhibitory receptor (IR), a TCR α (TRAC) constant region or a β chain (TRBC1/2) constant region target sequence, wherein said guide sequence is selected from the group consisting of SEQ ID NOs:1-27, 122-126 and combinations thereof.