44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Medical services, namely, cellular starter material collection services featuring human blood-derived biological raw materials that are eventually processed for biopharmaceutical uses and disease treatments
THERAPEUTIC ADENO-ASSOCIATED VIRUS USING CODON OPTIMIZED NUCLEIC ACID ENCODING ALPHA-GLUCOSIDASE (GAA) FOR TREATING POMPE DISEASE, WITH SIGNAL PEPTIDE MODIFICATIONS
Disclosed herein is a method for the treatment of Pompe Disease comprising administering a recombinant AAV (rAAV) vector comprising a rAVV genome comprising a heterologous nucleic acid encoding a GAA signal peptide or portion thereof, a heterologous signal peptide, and an acid alpha-glucosidase (GAA) polypeptide, or N-terminal truncation thereof, where the heterologous nucleic acid is operatively linked to a liver-specific promoter, where the nucleic acid encoding GAA polypeptide can be wild type nucleic acid sequence, or modified nucleic acid sequence, or a codon optimized nucleic acid sequence, and can optionally be modified to reduce or completely eliminate CG and CpG dinucleotides and, optionally eliminated alternative reading frames (ARF) content.
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 38/47 - Hydrolases (3) agissant sur des composés glycosyliques (3.2), p. ex. cellulases, lactases
A61P 3/00 - Médicaments pour le traitement des troubles du métabolisme
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Promoting the donation of blood plasma by means of arranging and conducting blood plasma donor loyalty and referral incentive rewards programs; administration of blood plasma donor loyalty and referral incentive rewards programs to promote blood plasma collection services and the collection of blood plasma; promoting public awareness of blood disorders, treatment and the impact of blood plasma collection. Providing a website promoting the donation of blood plasma by means of arranging and conducting blood plasma donor loyalty and referral incentive rewards programs; providing a website promoting blood plasma collection services and the collection of blood plasma; providing a website promoting public awareness of blood disorders, treatment and the impact of blood plasma collection.
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Promoting the donation of blood plasma by means of arranging and conducting blood plasma donor loyalty and referral incentive rewards programs; administration of blood plasma donor loyalty and referral incentive rewards programs to promote blood plasma collection services and the collection of blood plasma; promoting public awareness of blood disorders, treatment and the impact of blood plasma collection. Providing a website promoting the donation of blood plasma by means of arranging and conducting blood plasma donor loyalty and referral incentive rewards programs; providing a website promoting blood plasma collection services and the collection of blood plasma; providing a website promoting public awareness of blood disorders, treatment and the impact of blood plasma collection.
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
The present invention relates to regulatory nucleic acid sequences, in particular short liver-specific cis-regulatory elements, cis-regulatory modules, promoters and other such nucleic acid sequences, that are short and capable of enhancing liver-specific expression of genes. The invention also relates to expression constructs, vectors and cells comprising such short liver-specific regulatory nucleic acid sequences, and to methods of their use. The liver-specific regulatory nucleic acid sequences are of particular utility for gene therapy applications, but also find utility in other areas such as bioprocessing and biotechnology.
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 7/00 - Médicaments pour le traitement des troubles du sang ou du fluide extracellulaire
C12N 5/00 - Cellules non différenciées humaines, animales ou végétales, p. ex. lignées cellulairesTissusLeur culture ou conservationMilieux de culture à cet effet
15.
THERAPEUTIC PROTEINS WITH INCREASED HALF-LIFE AND METHODS OF PREPARING SAME
A61K 47/61 - 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 l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique macromoléculaire, p. ex. une molécule oligomérique, polymérique ou dendrimérique le composé organique macromoléculaire étant un polysaccharide ou l’un de ses dérivés
A61K 47/60 - 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 l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique macromoléculaire, p. ex. une molécule oligomérique, polymérique ou dendrimérique obtenu par des réactions autres que celles faisant intervenir uniquement des liaisons non saturées carbone-carbone, p. ex. polyurées ou polyuréthanes le composé organique macromoléculaire étant un oligomère, un polymère ou un dendrimère de polyoxyalkylène, p. ex. PEG, PPG, PEO ou polyglycérol
The present invention relates to regulatory nucleic acid sequences, in particular muscle- specific promoters, elements thereof, and other such nucleic acid sequences, that are capable of enhancing muscle-specific expression of genes. In particular, the invention relates to regulatory nucleic acid sequences capable of enhancing muscle-specific expression but having low liver-specific expression. The invention also relates to expression constructs, vectors and cells comprising such muscle-specific regulatory nucleic acid sequences, and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications, but also find utility in other areas such as bioprocessing and biotechnology.
09 - Appareils et instruments scientifiques et électriques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Non-downloadable software for patients, caregivers and
medical professionals for use in managing pharmaceutical
treatments and administering pharmaceutical preparations;
downloadable mobile applications for patients, caregivers,
and medical professionals for use in managing pharmaceutical
treatments and administering pharmaceutical preparations. Pharmaceutical patient support program providing guidance
and information on pharmaceutical treatment and
administration via non-downloadable software and a
downloadable mobile application.
09 - Appareils et instruments scientifiques et électriques
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
Downloadable software in the nature of a mobile application for patients, caregivers, and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations Providing online non-downloadable software for patients, caregivers and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations Pharmaceutical patient support program, namely, providing guidance and information to patients, caregivers, and medical professionals on pharmaceutical treatment and administration of pharmaceutical preparations via non-downloadable software and a downloadable mobile application
09 - Appareils et instruments scientifiques et électriques
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
Downloadable software in the nature of a mobile application for patients, caregivers, and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations Providing online non-downloadable software for patients, caregivers and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations Pharmaceutical patient support program, namely, providing guidance and information to patients, caregivers, and medical professionals on pharmaceutical treatment and administration of pharmaceutical preparations via non-downloadable software and a downloadable mobile application
09 - Appareils et instruments scientifiques et électriques
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
Downloadable software in the nature of a mobile application for patients, caregivers, and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations Providing online non-downloadable software for patients, caregivers and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations Pharmaceutical patient support program, namely, providing guidance and information to patients, caregivers, and medical professionals on pharmaceutical treatment and administration of pharmaceutical preparations via non-downloadable software and a downloadable mobile application
Educational materials and guides for school staff and administration to educate them on the unique needs and medical nuances of students living with bleeding conditions
09 - Appareils et instruments scientifiques et électriques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Non-downloadable software for patients, caregivers and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations; downloadable mobile applications for patients, caregivers, and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations. Pharmaceutical patient support program providing guidance and information on pharmaceutical treatment and administration via non-downloadable software and a downloadable mobile application.
29.
ADENOVIRUS-BASED NUCLEIC ACIDS AND METHODS THEREOF
C12N 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
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 reactor includes a support base, at least one wall, a top cover and a moveable plate assembly. The top cover, the support base and the at least one wall form an internal chamber therein. The moveable plate assembly includes a moveable plate located within the internal chamber. The moveable plate being configured to move in the x-direction, y-direction, and the z-direction. The temperature of the moveable plate is controllable to a desired temperature. The internal chamber is accessible via at least one of the top cover, the support base, and the at least one wall.
C12M 1/02 - Appareillage pour l'enzymologie ou la microbiologie avec des moyens d'agitationAppareillage pour l'enzymologie ou la microbiologie avec des moyens d'échange de chaleur
C12M 1/10 - Appareillage pour l'enzymologie ou la microbiologie montés rotativement
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
05 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
Chemical preparations, enzymes, DNA vectors, viral vectors, rAAV vectors, and DNA extracts for the preparation of nucleic acid, for scientific and research purposes; chemicals for use in the preparation and creation of DNA vectors, viral vectors, rAAV vectors, DNA extracts and cassettes for scientific and industrial purposes; enzymes for use in the preparation and creation of DNA extracts and cassettes for scientific and industrial purposes; DNA vectors, viral vectors, rAAV vectors, and DNA extracts and cassettes for scientific and research use; DNA vectors, viral vectors, rAAV vectors, DNA extracts for scientific and research purposes in the fields of agri-biotechnology and nanotechnology; Biological and biochemical reagents being DNA plasmid vectors, DNA vectors, and rAAV vectors, viral vectors that form a gene analysis system not for medical purposes; Diagnostic reagents for scientific or research purposes, namely, a diagnostic kit consisting primarily of DNA vectors, rAAV vectors, or DNA plasmid vectors. Vaccines; DNA based vaccines; DNA extracts, namely, nucleic acid sequences and chemical reagents for medical use; Therapeutic DNA, namely, nucleic acid sequences and chemical reagents for medical and veterinary purposes; Biotechnological products, namely, viral DNA vectors, DNA vectors, and rAAV vectors, for the delivery of genes and other materials, in the nature of DNA, RNA, or transcriptional targeting promoters, to a biological cell for medical purposes; Biological preparations, namely, viral vectors, rAAV vectors, and DNA extracts for production of DNA vectors and viral vectors, for medical purposes; Recombinant viruses. Scientific and technological services, namely, research and design in the field of DNA research and development; chemical and biological analysis and research services; scientific research in the field of DNA; scientific research and development in the field of creating DNA extracts; scientific and technological services, namely, scientific research, analysis, design and development in the field of DNA based pharmaceutical drugs and therapies; scientific research and development in the fields of gene therapy, diagnostics, agri-biotechnology and nanotechnology; scientific consultancy services in the fields of gene therapy, diagnostics, agri-biotechnology, nano technology and DNA analysis.
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
05 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
(1) Chemical preparations, enzymes, DNA vectors, viral vectors, rAAV vectors, and DNA extracts for the preparation of nucleic acid, for scientific and research purposes; chemicals for use in the preparation and creation of DNA vectors, viral vectors, rAAV vectors, DNA extracts and cassettes for scientific and industrial purposes; enzymes for use in the preparation and creation of DNA extracts and cassettes for scientific and industrial purposes; DNA vectors, viral vectors, rAAV vectors, and DNA extracts and cassettes for scientific and research use; DNA vectors, viral vectors, rAAV vectors, DNA extracts for scientific and research purposes in the fields of agri-biotechnology and nanotechnology; Biological and biochemical reagents being DNA plasmid vectors, DNA vectors, and rAAV vectors, viral vectors that form a gene analysis system not for medical purposes; Diagnostic reagents for scientific or research purposes, namely, a diagnostic kit consisting primarily of DNA vectors, rAAV vectors, or DNA plasmid vectors.
(2) Vaccines; DNA based vaccines; DNA extracts, namely, nucleic acid sequences and chemical reagents for medical use; Therapeutic DNA, namely, nucleic acid sequences and chemical reagents for medical and veterinary purposes; Biotechnological products, namely, viral DNA vectors, DNA vectors, and rAAV vectors, for the delivery of genes and other materials, in the nature of DNA, RNA, or transcriptional targeting promoters, to a biological cell for medical purposes; Biological preparations, namely, viral vectors, rAAV vectors, and DNA extracts for production of DNA vectors and viral vectors, for medical purposes; Recombinant viruses. (1) International Class 042: Scientific and technological services, namely, research and design in the field of DNA research and development; chemical and biological analysis and research services; scientific research in the field of DNA; scientific research and development in the field of creating DNA extracts; scientific and technological services, namely, scientific research, analysis, design and development in the field of DNA based pharmaceutical drugs and therapies; scientific research and development in the fields of gene therapy, diagnostics, agri-biotechnology and nanotechnology; scientific consultancy services in the fields of gene therapy, diagnostics, agri-biotechnology, nano technology and DNA analysis.
34.
FORSKOLIN-INDUCIBLE PROMOTERS AND HYPOXIA-INDUCIBLE PROMOTERS
Disclosed herein are codon-optimized nucleic acids encoding a Factor VIII polypeptide. Also disclosed are expression cassettes and expression vectors (e.g., recombinant AAV (rAAV) vectors) that contain the codon-optimized nucleic acids in expressible form. Methods for the treatment of Hemophilia A comprising administering expression vector comprising the codon-optimized nucleic acids (e.g., a recombinant AAV (rAAV) vector) are also disclosed.
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 7/04 - AntihémorragiquesProfacteurs de coagulationAgents hémostatiquesAgents antifibrinolytiques
A61K 31/711 - Acides désoxyribonucléiques naturels, c.-à-d. contenant uniquement des 2'-désoxyriboses liés à l'adénine, la guanine, la cytosine ou la thymine et ayant des liaisons 3'-5' phosphodiester
The present invention relates to regulatory nucleic acid sequences, in particular NS-specific promoters, cell specific promoters, multi-cell specific promoters, and elements thereof. The invention also relates to expression constructs, vectors, virions, pharmaceutical compositions and cells comprising such promoters and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
42 - Services scientifiques, technologiques et industriels, recherche et conception
45 - Services juridiques; services de sécurité; services personnels pour individus
Produits et services
Promoting collaboration between scientists, technicians, clinical specialists, patients, and their families to achieve advances in therapeutics, technology, regulatory requirements, and public policy in the field of healthcare, genetic disease, gene therapy, and clinical trials Providing medical and scientific research information in the field of gene therapy, and clinical trials; consulting services for others in the field of designing clinical trials; medical and scientific research, namely, designing clinical trials for others Providing patient advocate services in the field of genetic disease, gene therapy, and potential curative therapies; providing personal support services for patients, namely, emotional counseling and emotional support by providing personal emotional support information in the fields of genetic disease, gene therapy, and potential curative therapies; providing patient advocacy and personal emotional support services to potential participants and participants in clinical trials for genetic diseases and gene therapy; providing information in the field of personal emotional support of gene therapy patients and patients' families
38.
METHODS AND COMPOSITIONS FOR THE TREATMENT OF PARKINSON'S DISEASE
Aspects of the disclosure relate to compositions and methods useful for treating Parkinson's disease. In some embodiments, the disclosure provides a method for treating Parkinson's disease comprising administration of a viral vector comprising a GDNF nucleic acid sequence. In some embodiments, administration is locally to the subject putamen. In some embodiments, administration is systemically, e.g., via the viral vector comprising a modified viral capsid, such as for preferentially targeting cells in the CNS or PNS.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
THERAPEUTIC ADENO-ASSOCIATED VIRUS USING CODON OPTIMIZED NUCLEIC ACID ENCODING ALPHA-GLUCOSIDASE (GAA) FOR TREATING POMPE DISEASE, WITH SIGNAL PEPTIDE MODIFICATIONS
Disclosed herein is a method for the treatment of Pompe Disease comprising administering a recombinant AAV (rAAV) vector comprising a rAVV genome comprising a heterologous nucleic acid encoding a GAA signal peptide or portion thereof, a heterologous signal peptide, and an acid alpha-glucosidase (GAA) polypeptide, or N-terminal truncation thereof, where the heterologous nucleic acid is operatively linked to a liver-specific promoter, where the nucleic acid encoding GAA polypeptide can be wild type nucleic acid sequence, or modified nucleic acid sequence, or a codon optimized nucleic acid sequence, and can optionally be modified to reduce or completely eliminate CG and CpG dinucleotides and, optionally eliminated alternative reading frames (ARF) content.
A61K 38/47 - Hydrolases (3) agissant sur des composés glycosyliques (3.2), p. ex. cellulases, lactases
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 9/26 - Hydrolases (3.) agissant sur les composés glycosyliques (3.2) agissant sur les liaisons alpha-glucosidiques-1, 4, p. ex. hyaluronidase, invertase, amylase
C12N 15/12 - Gènes codant pour des protéines animales
09 - Appareils et instruments scientifiques et électriques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Downloadable mobile application for patients, caregivers, and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations. Pharmaceutical patient support program providing guidance and information on pharmaceutical treatment and administration via a downloadable mobile application.
09 - Appareils et instruments scientifiques et électriques
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Downloadable mobile application for patients, caregivers, and medical professionals for use in managing pharmaceutical treatments and administering pharmaceutical preparations. Pharmaceutical patient support program providing guidance and information on pharmaceutical treatment and administration via a downloadable mobile application.
42.
THERAPEUTIC ADENO-ASSOCIATED VIRUS FOR TREATING POMPE DISEASE WITH LONG TERM CESSATION OF GAA ENZYME REPLACEMENT THERAPY
Disclosed herein are methods for the treatment of Pompe Disease comprising administering a recombinant AAV (rAAV) vector comprising a rAVV genome comprising a heterologous nucleic acid encoding an acid alpha-glucosidase (GAA) polypeptide operatively linked to a liver-specific promoter, wherein the subject is withdrawn or not administered enzyme replacement therapy (ERT).
A61K 38/47 - Hydrolases (3) agissant sur des composés glycosyliques (3.2), p. ex. cellulases, lactases
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 3/00 - Médicaments pour le traitement des troubles du métabolisme
Aspects of the disclosure relate to compositions and methods useful for treating neurological diseases and disorders. In some embodiments, the disclosure provides a method for treating a neurological disease or disorder comprising administration of both a viral vector comprising interfering nucleic acids (e.g., artificial miRNAs) and a viral vector comprising a CYP46A1 protein. In some embodiments, the disclosure provides a method for treating Huntington's disease comprising administration of both a viral vector comprising interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and a viral vector comprising a CYP46A1 protein. In some embodiments, the viral vector comprises a modified viral capsid, such as for preferentially targeting cells in the CNS or PNS.
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
A61P 25/00 - Médicaments pour le traitement des troubles du système nerveux
The present invention relates to regulatory nucleic acid sequences, in particular muscle-specific promoters, elements thereof, and other such nucleic acid sequences, that are capable of enhancing muscle-specific expression of genes. The invention also relates to expression constructs, vectors and cells comprising such muscle-specific regulatory nucleic acid sequences, and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications, but also find utility in other areas such as bioprocessing and biotechnology.
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 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
Printed educational materials and guides for school staff and administration to educate them on the unique needs and medical nuances of students living with bleeding conditions
46.
FORSKOLIN-INDUCIBLE PROMOTERS AND HYPOXIA-INDUCIBLE PROMOTERS
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
Scientific and technological services, namely, research and design in the field of DNA research and development; chemical and biological analysis and research services; scientific research in the field of DNA; scientific research and development in the field of creating DNA extracts; scientific and technological services, namely, scientific research, analysis, design and development in the field of DNA based pharmaceutical drugs and therapies; scientific research and development in the fields of gene therapy, diagnostics, agri-biotechnology and nanotechnology; scientific consultancy services in the fields of gene therapy, diagnostics, agri-biotechnology, nano technology and DNA analysis
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
05 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
DNA vectors, viral vectors, rAAV vectors, and DNA extracts for the preparation of nucleic acid for scientific and research purposes; chemicals for use in the preparation and creation of DNA vectors, viral vectors, rAAV vectors, DNA extracts and cassettes for scientific and industrial purposes; enzymes for use in the preparation and creation of DNA vectors, rAAV vectors, DNA extracts and cassettes for scientific and industrial purposes; DNA vectors, viral vectors, rAAV vectors, and DNA extracts and cassettes for scientific and research use; DNA vectors, viral vectors, rAAV vectors, and DNA extracts for scientific and research purposes in the fields of agri-biotechnology and nanotechnology; Biological and biochemical reagents being RNA polymerase, DNA polymerase, DNA plasmid vectors, DNA vectors, viral vectors, and rAAV vectors, and RNA markers that form a gene analysis system not for medical purposes; Diagnostic reagents for scientific or research purposes, namely, a diagnostic kit consisting primarily of DNA vectors, rAAV vectors, or DNA plasmid vectors. Vaccines; DNA based vaccines; DNA extracts, namely, nucleic acid sequences and chemical reagents for medical use; Therapeutic DNA, namely, nucleic acid sequences and chemical reagents for medical and veterinary purposes; Biotechnological products, namely, viral DNA vectors, DNA vectors, and rAAV vectors, for the delivery of genes and other materials, in the nature of DNA, RNA, or transcriptional targeting promoters, to a biological cell for medical purposes; Biological preparations, namely, viral vectors, rAAV vectors, and DNA extracts for production of DNA vectors and viral vectors for medical purposes; Recombinant viruses. Scientific and technological services, namely, research and design in the field of DNA research and development; chemical and biological analysis and research services; scientific research in the field of DNA; scientific research and development in the field of creating DNA extracts; scientific and technological services, namely, scientific research, analysis, design and development in the field of DNA based pharmaceutical drugs and therapies; scientific research and development in the fields of gene therapy, diagnostics, agri-biotechnology and nanotechnology; scientific consultancy services in the fields of gene therapy, diagnostics, agri-biotechnology, nano technology, and DNA analysis.
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
05 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
Chemical preparations, enzymes, DNA vectors, viral vectors, rAAV vectors, and DNA extracts for the preparation of nucleic acid for scientific and research purposes; chemicals for use in the preparation and creation of DNA vectors, viral vectors, rAAV vectors, DNA extracts and cassettes for scientific and industrial purposes; enzymes for use in the preparation and creation of DNA vectors, rAAV vectors, DNA extracts and cassettes for scientific and industrial purposes; DNA vectors, viral vectors, rAAV vectors, and DNA extracts and cassettes for scientific and research use; DNA vectors, viral vectors, rAAV vectors, and DNA extracts for scientific and research purposes in the fields of agri-biotechnology and nanotechnology; Biological and biochemical reagents being RNA polymerase, DNA polymerase, DNA plasmid vectors, DNA vectors, viral vectors, and rAAV vectors, and RNA markers that form a gene analysis system not for medical purposes; Diagnostic reagents for scientific or research purposes, namely, a diagnostic kit consisting primarily of DNA vectors, rAAV vectors, or DNA plasmid vectors. Vaccines; DNA based vaccines; DNA extracts, namely, nucleic acid sequences and chemical reagents for medical use; Therapeutic DNA, namely, nucleic acid sequences and chemical reagents for medical and veterinary purposes; Biotechnological products, namely, viral DNA vectors, DNA vectors, and rAAV vectors, for the delivery of genes and other materials, in the nature of DNA, RNA, or transcriptional targeting promoters, to a biological cell for medical purposes; Biological preparations, namely, viral vectors, rAAV vectors, and DNA extracts for production of DNA vectors and viral vectors for medical purposes; Recombinant viruses. Scientific and technological services, namely, research and design in the field of DNA research and development; chemical and biological analysis and research services; scientific research in the field of DNA; scientific research and development in the field of creating DNA extracts; scientific and technological services, namely, scientific research, analysis, design and development in the field of DNA based pharmaceutical drugs and therapies; scientific research and development in the fields of gene therapy, diagnostics, agri-biotechnology and nanotechnology; scientific consultancy services in the fields of gene therapy, diagnostics, agri-biotechnology, nano technology, and DNA analysis.
The present invention relates to regulatory nucleic acid sequences, in particular CNS—specific promoters, and elements thereof. The invention also relates to expression constructs, vectors, virions, pharmaceutical compositions and cells comprising such promoters and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications.
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
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Vaccines; DNA based vaccines; DNA extracts, namely, nucleic acid sequences and chemical reagents for medical use; Therapeutic DNA, namely, nucleic acid sequences and chemical reagents for medical and veterinary purposes; Biotechnological products, namely, viral DNA vectors, DNA vectors, and rAAV vectors, for the delivery of genes and other materials, in the nature of DNA, RNA, and transcriptional targeting promoters, to a biological cell for medical purposes; Biological preparations, namely, viral vectors, rAAV vectors, and DNA extracts for production of DNA vectors and viral vectors for medical purposes
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Vaccines; DNA based vaccines; DNA extracts, namely, nucleic acid sequences and chemical reagents for medical use; Therapeutic DNA, namely, nucleic acid sequences and chemical reagents for medical and veterinary purposes; Biotechnological products, namely, viral DNA vectors, DNA vectors, and rAAV vectors, for the delivery of genes and other materials, in the nature of DNA, RNA, and transcriptional targeting promoters, to a biological cell for medical purposes; Biological preparations, namely, viral vectors, rAAV vectors, and DNA extracts for production of DNA vectors and viral vectors for medical purposes
Recombinant AAV (rAAV) vectors comprising a rAVV genome comprising a heterologous nucleic acid encoding a lysosomal protein, e.g., acid alpha-glucosidase (GAA) polypeptide, and optionally a signal peptide and/or optionally a targeting sequence, e.g., IGF2 targeting peptide, operatively linked to a liver-specific promoter (LSP), enabling the GAA polypeptide to be secreted from the liver and targeted to the lysosomes. Particular embodiments relate to a recombinant AAV (rAAV) vector encoding an alpha-glucosidase (GAA) polypeptide, having a liver secretory signal peptide and a IGF2 targeting peptide that binds human cation-independent mannose-6-phosphate receptor (CI-MPR) or to the IGF2 receptor, permitting proper subcellular localization of the GAA polypeptide to lysosomes. Also encompassed are cells, and methods to treat a lysosomal disease, for example, a glycogen storage disease type II (GSD II) disease and/or Pompe Disease with the rAAV vector.
C12N 9/26 - Hydrolases (3.) agissant sur les composés glycosyliques (3.2) agissant sur les liaisons alpha-glucosidiques-1, 4, p. ex. hyaluronidase, invertase, amylase
The present invention relates to regulatory nucleic acid sequences, in particular muscle- specific promoters, elements thereof, and other such nucleic acid sequences, that are capable of enhancing muscle-specific expression of genes. The invention also relates to expression constructs, vectors and cells comprising such muscle-specific regulatory nucleic acid sequences, and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications, but also find utility in other areas such as bioprocessing and biotechnology.
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 cannula assembly includes a cannula, polymeric support material and a hub. The cannula has a proximal end and a distal end. The polymeric support material substantially surrounds a portion of the cannula at or near the proximal end. The hub is configured to be attached to a syringe. The polymeric support material is located between the cannula and the hub. The cannula, the polymeric support material and the hub are adhesively attached.
The present invention relates to a substantially homogenous population of a rational polyploid adeno-associated virus (AAV) virons that cross the blood brain barrier (BBB), where the rational polyploid comprises a VP3 viral structural protein from any AAV serotype that cross the BBB. In some embodiments, the rational polyploid crosses the BBB upon systemic or intrathecal administration to a subject. In some embodiments, a rational polyploid AAV virion comprises at least one VP1 and/or VP2 viral structural protein in addition to the VP3 protein. In some embodiments, the VP3 capsid protein is from a non-human primate, and in some embodiments the VP3 capsid protein is a AAV rhesus monkey serotype. In specific embodiments, rational polyploid AAV virion comprises a VP1 capsid protein from AAV8, and at least a VP3 capsid protein from any AAV serotype that cross the BBB.
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
Produits et services
(1) Produits pharmaceutiques et vétérinaires ainsi que produits hygiéniques, à savoir, préparations pharmaceutiques pour la prévention et le traitement des saignements causés par des troubles sanguins; produits diététiques pour enfants et malades, à savoir, suppléments alimentaires pour l'état général de la santé et le bien-être; emplâtres en tant que matériel pour pansements, matériel pour pansements, à savoir, pansements médicaux; matières pour plomber les dents et pour empreintes dentaires; désinfectants multi usages; préparations pour détruire les mauvaises herbes et les animaux nuisibles.
Provided herein are purification, production and manufacturing methods for recombinant viral vector particles such as recombinant adeno-associated viral (rAAV) vector particles substantially free of empty viral particles; a population of recombinant adeno-associated virus (rAAV) particles purified using the method described herein, and a pharmaceutical composition comprising the purified rAAV.
B01D 15/38 - Adsorption sélective, p. ex. chromatographie caractérisée par le mécanisme de séparation impliquant une interaction spécifique non couverte par un ou plusieurs des groupes , p. ex. chromatographie d'affinité, chromatographie d'échange par ligand ou chromatographie chirale
B01D 15/36 - Adsorption sélective, p. ex. chromatographie caractérisée par le mécanisme de séparation impliquant une interaction ionique, p. ex. échange d'ions, paire d'ions, suppression d'ions ou exclusion d'ions
B01D 15/16 - Adsorption sélective, p. ex. chromatographie caractérisée par des caractéristiques de structure ou de fonctionnement relatives au conditionnement du fluide vecteur
B01D 15/42 - Adsorption sélective, p. ex. chromatographie caractérisée par le mode de développement, p. ex. par déplacement ou par élution
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
Produits et services
Chemical preparations, enzymes, DNA vectors, and DNA extracts for the preparation of nucleic acid for scientific and research purposes; chemicals for use in the preparation and creation of DNA extracts and cassettes for scientific and industrial purposes; enzymes for use in the preparation and creation of DNA extracts and cassettes for scientific and industrial purposes; DNA extracts and cassettes for scientific and research use; DNA extracts for scientific and research purposes in the fields of agri-biotechnology and nanotechnology; biological and biochemical reagents being RNA polymerase, DNA polymerase, DNA plasmid vectors, DNA vectors, rAAV vectors, and RNA markers that form a gene analysis system not for medical purposes; diagnostic reagents for scientific or research purposes, namely, a diagnostic kit consisting primarily of DNA plasmid vectors
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
05 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
Chemical preparations, enzymes, DNA vectors, and DNA extracts for the preparation of nucleic acid for scientific and research purposes; chemicals for use in the preparation and creation of DNA extracts and cassettes for scientific and industrial purposes; enzymes for use in the preparation and creation of DNA extracts and cassettes for scientific and industrial purposes; DNA extracts and cassettes for scientific and research use; DNA extracts for scientific and research purposes in the fields of agri-biotechnology and nanotechnology; Biological and biochemical reagents being RNA polymerase, DNA polymerase, DNA plasmid vectors, DNA vectors, rAAV vectors, and RNA markers that form a gene analysis system not for medical purposes; Diagnostic reagents for scientific or research purposes, namely, a diagnostic kit consisting primarily of DNA plasmid vectors Vaccines; DNA based vaccines; DNA extracts, namely, nucleic acid sequences and chemical reagents for medical use; Therapeutic DNA, namely, nucleic acid sequences and chemical reagents for medical and veterinary purposes; Biotechnological product, namely, viral DNA vectors, for the delivery of genes and other materials in the nature of RNA and transcriptional targeting promoters, to a biological cell for medical purposes; biological preparations, namely, DNA extracts for production of viral vectors for medical purposes Scientific and technological services, namely, research and design in the field of DNA research and development; chemical and biological analysis and research services; scientific research in the field of DNA; scientific research and development in the field of creating DNA extracts; scientific and technological services, namely, scientific research, analysis, design and development in the field of DNA based pharmaceutical drugs and therapies; scientific research and development in the fields of gene therapy, diagnostics, agri-biotechnology and nanotechnology; scientific consultancy services in the fields of gene therapy, diagnostics, agri-biotechnology, nano technology, and DNA analysis
THE UNIVERSITY OF NORTH CAROLINA CHAPEL HILL (USA)
Inventeur(s)
Childers, Martin K.
Yanez-Cuna, Jorge Omar
Iglesias Gonzalez, Juan Manuel
Cooper, Sinclair
Evripioti, Antonia
Roberts, Michael L.
Tretiakova, Anna
Suarez, Lester
Nguyen, Anh
Low, Siewhui
Xiao, Xiao
Abrégé
Disclosed herein are various optimized nucleic acids encoding the fukutin-related protein (FKRP). Recombinant vectors comprising the optimized nucleic acid (e.g. operatively linked to a muscle specific promoter), such as recombinant adeno-associated virus vectors, for expressing the protein (e.g. in skeletal and cardiac muscle), and therapeutic compositions contains the vectors are also disclosed. Therapeutic methods of administration of the vectors to a subject for the treatment of a subject with a dystroglycanopathy disorder (e.g., limb-girdle muscular dystrophy 2I) are also disclosed.
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 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
Medical devices, namely, vial access system consisting primarily of a docking tray that facilitates direct access to contents of multiple vials containing pharmaceutical preparations for patient infusion.
Medical devices, namely, vial access system consisting primarily of a docking tray that facilitates direct access to contents of multiple vials containing pharmaceutical preparations for patient infusion
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et services
Medical services, namely, cellular starter material collection services featuring human blood-derived biological raw materials that are eventually processed for biopharmaceutical uses and disease treatments
Aspects of the disclosure relate to compositions and methods useful for treating neurological diseases and disorders. In some embodiments, the disclosure provides a method for treating a neurological disease or disorder comprising administration of both a viral vector comprising interfering nucleic acids (e.g., artificial miRNAs) and a viral vector comprising a CYP46A1 protein. In some embodiments, the disclosure provides a method for treating Huntington's disease comprising administration of both a viral vector comprising interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and a viral vector comprising a CYP46A1 protein. In some embodiments, the viral vector comprises a modified viral capsid, such as for preferentially targeting cells in the CNS or PNS.
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
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 regulatory nucleic acid sequences, in particular NS-specific promoters, cell specific promoters, multi-cell specific promoters, and elements thereof. The invention also relates to expression constructs, vectors, virions, pharmaceutical compositions and cells comprising such promoters and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications.
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 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
81.
METHODS OF TREATING CARDIAC DISORDERS AND CONGESTIVE HEART FAILURE AND ADMINISTERING AAV VECTORS
The present invention relates to methods of administration of rAAV vectors in a single administration method comprising a series of sub-administrations of sub-doses of rAAV vectors. The present invention also relates to rAAV vectors comprising cardiac-specific promoters, cardiac-cell specific promoters, multi-cell cardiac specific promoters, and elements thereof. The invention also relates to rAAV vectors, pharmaceutical compositions and uses thereof in methods for treating cardiovascular disease, heart diseases and heart failure in subjects 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
A liquid handling robot has a worktable that supports a rack holding a set of pipette tips. The liquid handling robot also has an arm that is operably suspended above the worktable, where the arm includes a tip receiver that is configured to engage the set of pipette tips. A controller of the liquid handling robot is configured to raise the tip receiver away from the worktable to withdraw the engaged set of pipette tips from the rack. A sensor is fixed relative to the worktable and is operable to emit a beam. A microcontroller monitors the sensor with the arm in a checking position to determine if the rack interrupts in the beam to indicate that the rack stuck to the pipette tips, which is autonomously resolved by the liquid handling robot performing a corrective action.
A filter press device includes a supporting frame and elongated side rails disposed parallel and horizontally on the supporting frame. A following head assembly is disposed on an end portion of the supporting frame, and a following head assembly is movably disposed on another end portion of the supporting frame. The elongated side rails support a combination of filter plates of varying sizes through one or more adapters. An adapter plate interposes between adjacent filter plates of different sizes, providing fluidic communication between the different sized filter plates.
B01D 25/164 - Presses à chambre et à plaque, c.-à-d. dans lesquelles les bords latéraux des éléments filtrants sont calés entre les deux plaques filtrantes successives
A medicinal fluid pooling device may be used to pool multiple containers of medicinal fluid to facilitate administration of the medicinal fluid to a patient. A medicinal pooling device may include spikes covered by spike sheaths which are pierced when a container of medicinal fluid is inserted into the medicinal pooling device. The medicinal pooling device may also include a cover configured to cover the spikes. The medicinal pooling device may also include a fluidic interface which may be used to fluidly connect the medicinal pooling device to an infusion pump or syringe.
A61M 5/162 - Raccords à aiguilles, c.-à-d. raccordements par perforation entre le réservoir et le tube
A61M 5/14 - Dispositifs de perfusion, p. ex. perfusion par gravitéPerfusion sanguineAccessoires à cet effet
A61M 5/168 - Moyens pour commander l'écoulement des agents vers le corps ou pour doser les agents à introduire dans le corps, p. ex. compteurs de goutte-à-goutte
The present invention relates to regulatory nucleic acid sequences, in particular CNS- specific promoters, and elements thereof. The invention also relates to expression constructs, vectors, virions, pharmaceutical compositions and cells comprising such promoters and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications.
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
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 15/79 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes
88.
A METHOD TO PRODUCE AN IMMUNOGLOBULIN PREPARATION FROM C-1 INHIBITOR DEPLETED PLASMA
Described is a method for preparing an Immunoglobulin G (IgG) enriched fraction from a Cl- INH depleted plasma supernatant. Isolation of Immunoglobulin G (IgG) enriched fraction from a Cl-INH depleted plasma supernatant provided an alternative starting material for the manufacturing process. In the present invention, Cl-INH depleted plasma supernatant is treated with heparin before further processing.
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
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 15/79 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes
Aspects described herein relate to stable cell line for recombinant viral vector (e.g., recombinant adeno associated viral vector) production comprising at least one inducible promoter operatively linked to a heterologous gene that encodes a toxic protein. Methods for making stable cell lines, and methods for viral vector production are further provided herein.
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
Aspects described herein relate to stable cell line for recombinant viral vector (e.g., recombinant adeno associated viral vector) production comprising at least one inducible promoter operatively linked to a heterologous gene that encodes a toxic protein. Methods for making stable cell lines, and methods for viral vector production are further provided herein.
In some embodiments, a reconstitution or medicinal fluid delivery device includes a transfer engine including two fluidly connected spikes, each configured to pierce a container. A check valve may be disposed between the two spikes to allow unidirectional flow from one container to the other. Physical access to a fluid outlet may be obstructed by a housing until the reconstitution or medicinal fluid delivery device is actuated, whereupon physical access to the fluid outlet is permitted. The reconstitution or medicinal fluid delivery device may be placed on a flat surface and actuated with force applied in a single direction toward the flat surface.
Disclosed herein are methods of treating a toxicity in a subject receiving recombinant viral vector, such as a recombinant adeno-associated viral AAV (rAAV) vector comprising co-administration of an antibiotic and a viral vector. In one embodiment, the antibiotic is a tetracycline or macrolide family member. Also provided herein are compositions comprising an antibiotic and a viral vector.
The present invention relates to a method for treatment of menorrhagia in a subject with von Willebrand Disease (VWD) comprising administering a therapeutic amount of recombinant von Willebrand Factor (rVWF) to the subject.
The present invention provides, among other aspects, methods for the treatment of Alzheimer's disease in a subject in need thereof, the method including administration of a therapeutically effective amount of a pooled human immunoglobulin G (IgG) composition to a subject with moderately severe Alzheimer's disease, a subject carrying an ApoE4 allele, or both, where the amount of pooled human IgG is from 300 mg/kg to 800 mg/kg body weight of the subject per two week period, and where the amount is administered in one or more doses during the two week period after initiation of a therapeutic regimen. Also provided, are methods for selecting a treatment regimen for a subject with Alzheimer's disease, including diagnosing the severity of the Alzheimer's disease, determining if the subject carries an APOE4 allele, or both, and assigning a treatment regimen including administration of pooled human immunoglobulin G and/or an anti-beta amyloid monoclonal antibody.
C07K 16/18 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains
A61K 38/47 - Hydrolases (3) agissant sur des composés glycosyliques (3.2), p. ex. cellulases, lactases
G01N 33/68 - Analyse chimique de matériau biologique, p. ex. de sang ou d'urineTest par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligandsTest immunologique faisant intervenir des protéines, peptides ou amino-acides
A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
C07K 16/06 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux provenant de sérum
C07K 16/00 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux
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
97.
SYSTEMS AND METHODS FOR SYNTHETIC REGULATORY SEQUENCE DESIGN OR PRODUCTION
The technology described herein is directed to systems and methods for synthetic regulatory sequence design or production. In several aspects described herein are methods of designing and optionally synthesizing shortened or otherwise modified polynucleotide sequences from polynucleotide sequences with transcriptional regulatory functionality. In other aspects described herein are isolated nucleic acid modules and viral vectors, comprising said shortened or otherwise modified polynucleotide as designed or synthesized herein.
G06F 19/18 - pour la génomique ou la protéomique fonctionnelle, p.ex. associations génotype-phénotype, déséquilibre de liaison, mutagénèse, génotypage ou annotation génomique, interactions protéines-protéines ou interactions protéines-acides nucléiques
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
C12N 15/88 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation utilisant la micro-encapsulation, p. ex. utilisant des vésicules liposomiques
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
C12N 15/87 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation
C12Q 1/70 - 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 virus ou des bactériophages
99.
METHOD FOR IDENTIFYING REGULATORY ELEMENTS CONFORMATIONALLY
The present invention provides a method of identifying the strength of one or more unique regulatory elements (URE) having conformational effect on a transcribable reporter sequence.
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C12N 15/79 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
C12N 15/00 - Techniques de mutation ou génie génétiqueADN ou ARN concernant le génie génétique, vecteurs, p. ex. plasmides, ou leur isolement, leur préparation ou leur purificationUtilisation d'hôtes pour ceux-ci
The present invention relates to regulatory nucleic acid sequences, in particular muscle- specific promoters, elements thereof, and other such nucleic acid sequences, that are capable of enhancing muscle-specific expression of genes. The invention also relates to expression constructs, vectors and cells comprising such muscle-specific regulatory nucleic acid sequences, and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications, but also find utility in other areas such as bioprocessing and biotechnology.
