CA3096088A1 - Compositions and methods for treating macular dystrophy - Google Patents
Compositions and methods for treating macular dystrophy Download PDFInfo
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Classifications
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0083—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the administration regime
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0048—Eye, e.g. artificial tears
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
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Landscapes
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- Chemical & Material Sciences (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Manufacturing & Machinery (AREA)
- Dermatology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
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| US201862653131P | 2018-04-05 | 2018-04-05 | |
| US62/653,131 | 2018-04-05 | ||
| PCT/US2019/026062 WO2019195727A1 (en) | 2018-04-05 | 2019-04-05 | Compositions and methods for treating macular dystrophy |
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| CA3096088A1 true CA3096088A1 (en) | 2019-10-10 |
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|---|---|---|---|
| CA3096088A Withdrawn CA3096088A1 (en) | 2018-04-05 | 2019-04-05 | Compositions and methods for treating macular dystrophy |
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| EP (1) | EP3775233A1 (enExample) |
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| SG (1) | SG11202009759SA (enExample) |
| TW (1) | TW202003052A (enExample) |
| WO (1) | WO2019195727A1 (enExample) |
Cited By (1)
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| CN111849998A (zh) * | 2020-07-29 | 2020-10-30 | 武汉纽福斯生物科技有限公司 | 编码人卵黄状黄斑病蛋白1的核酸分子及其应用 |
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| WO2020047476A1 (en) * | 2018-08-31 | 2020-03-05 | University Of Florida Research Foundation, Incorporated | Adeno-associated viral vectors for the treatment of best disease |
| EP3870148A4 (en) | 2018-10-25 | 2022-11-09 | Takeda Pharmaceutical Company Limited | AAV TRIPLE PLASMID SYSTEM |
| WO2020140007A1 (en) * | 2018-12-28 | 2020-07-02 | University Of Rochester | Gene therapy for best1 dominant mutations |
| BR112022017182A2 (pt) * | 2020-02-28 | 2022-11-08 | Univ Pennsylvania | Tratamento de bestrofinopatias autossômicas recessivas e métodos para avaliação das mesmas |
| WO2024064608A2 (en) * | 2022-09-20 | 2024-03-28 | Intergalactic Therapeutics, Inc. | Best1 vectors and uses thereof |
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| WO2009153563A1 (en) * | 2008-06-18 | 2009-12-23 | Oxford Biomedica (Uk) Limited | Virus purification |
| US9163259B2 (en) * | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
| CA2930282C (en) * | 2013-11-20 | 2024-09-10 | Universita Degli Studi Di Napoli Federico Ii | Artificial DNA-Binding Proteins and Their Uses |
| KR102281881B1 (ko) * | 2013-12-06 | 2021-07-27 | 엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔 (인쎄름) | 대상체의 망막 색소 상피에서 목적 폴리뉴클레오티드를 발현시키기 위한 방법 및 약학적 조성물 |
| GB201322798D0 (en) * | 2013-12-20 | 2014-02-05 | Oxford Biomedica Ltd | Production system |
| CA2979120A1 (en) * | 2015-03-03 | 2016-09-09 | Fondazione Telethon | Multiple aav vector system for expressing genes over 5 kilobases, comprising at least one degradation signal and uses thereof |
| EP3374494A4 (en) * | 2015-11-11 | 2019-05-01 | Coda Biotherapeutics, Inc. | CRISPR COMPOSITIONS AND METHODS OF USE THEREOF FOR GENE THERAPY |
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Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN111849998A (zh) * | 2020-07-29 | 2020-10-30 | 武汉纽福斯生物科技有限公司 | 编码人卵黄状黄斑病蛋白1的核酸分子及其应用 |
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| EP3775233A1 (en) | 2021-02-17 |
| AU2019247864A1 (en) | 2020-10-22 |
| WO2019195727A1 (en) | 2019-10-10 |
| MA52199A (fr) | 2021-02-17 |
| BR112020020204A2 (pt) | 2021-01-19 |
| PE20210918A1 (es) | 2021-05-19 |
| US20190307900A1 (en) | 2019-10-10 |
| JP2021520232A (ja) | 2021-08-19 |
| IL277779A (en) | 2020-11-30 |
| PH12020551641A1 (en) | 2021-07-26 |
| US20230149566A1 (en) | 2023-05-18 |
| MX2020010477A (es) | 2021-03-02 |
| JOP20200253A1 (ar) | 2020-10-04 |
| CL2020002561A1 (es) | 2021-04-23 |
| SG11202009759SA (en) | 2020-10-29 |
| CN113056561A (zh) | 2021-06-29 |
| RU2020132890A (ru) | 2022-05-06 |
| KR20210005040A (ko) | 2021-01-13 |
| TW202003052A (zh) | 2020-01-16 |
| CO2020013690A2 (es) | 2021-04-19 |
| EA202092069A1 (ru) | 2021-03-12 |
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| Date | Code | Title | Description |
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| AZWI | Withdrawn application |
Effective date: 20230718 |