RU2019100525A3 - - Google Patents
Download PDFInfo
- Publication number
- RU2019100525A3 RU2019100525A3 RU2019100525A RU2019100525A RU2019100525A3 RU 2019100525 A3 RU2019100525 A3 RU 2019100525A3 RU 2019100525 A RU2019100525 A RU 2019100525A RU 2019100525 A RU2019100525 A RU 2019100525A RU 2019100525 A3 RU2019100525 A3 RU 2019100525A3
- Authority
- RU
- Russia
Links
Classifications
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
-
- 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
-
- 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
- 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
- C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
-
- 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
-
- 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
- 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
- C12N2750/14171—Demonstrated in vivo effect
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Virology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Ophthalmology & Optometry (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Cell Biology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- General Chemical & Material Sciences (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Mycology (AREA)
- Toxicology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GB1610448.1 | 2016-06-15 | ||
| GBGB1610448.1A GB201610448D0 (en) | 2016-06-15 | 2016-06-15 | Adeno-associated viral vector system |
| GB1707261.2 | 2017-05-05 | ||
| GBGB1707261.2A GB201707261D0 (en) | 2017-05-05 | 2017-05-05 | Adeno-associated viral vector system |
| PCT/GB2017/051741 WO2017216560A1 (en) | 2016-06-15 | 2017-06-14 | Dual overlapping adeno-associated viral vector system for expressing abc4a |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| RU2019100525A RU2019100525A (ru) | 2020-07-15 |
| RU2019100525A3 true RU2019100525A3 (enExample) | 2020-07-15 |
| RU2765826C2 RU2765826C2 (ru) | 2022-02-03 |
Family
ID=59101505
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2019100525A RU2765826C2 (ru) | 2016-06-15 | 2017-06-14 | Векторная система на основе аденоассоциированного вируса |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20190309326A1 (enExample) |
| EP (1) | EP3472328A1 (enExample) |
| JP (1) | JP2019523648A (enExample) |
| KR (1) | KR20190020745A (enExample) |
| CN (1) | CN109642242A (enExample) |
| AU (1) | AU2017286623A1 (enExample) |
| BR (1) | BR112018075855A2 (enExample) |
| CA (1) | CA3025445A1 (enExample) |
| IL (1) | IL263523A (enExample) |
| MX (1) | MX2018015629A (enExample) |
| RU (1) | RU2765826C2 (enExample) |
| SG (1) | SG11201811244SA (enExample) |
| WO (1) | WO2017216560A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2986635T3 (en) | 2013-04-18 | 2019-01-28 | Fond Telethon | EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS |
| US12359221B2 (en) | 2017-05-05 | 2025-07-15 | The Regents Of The University Of California | Compositions and methods for expressing otoferlin |
| US20210147870A1 (en) * | 2018-04-05 | 2021-05-20 | Nightstarx Limited | Compositions and methods for the treatment of stargardt disease |
| CN113348249B (zh) * | 2018-10-15 | 2025-09-23 | 特莱索恩基金会 | 内蛋白及其用途 |
| GB201817469D0 (en) * | 2018-10-26 | 2018-12-12 | Univ Oxford Innovation Ltd | Gene therapy for retinal disease |
| WO2020093018A1 (en) | 2018-11-01 | 2020-05-07 | University Of Florida Research Foundation, Incorporated | A codon optimized otoferlin aav dual vector gene therapy |
| EP3921032A4 (en) | 2019-02-08 | 2022-11-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| US20220267802A1 (en) * | 2019-07-15 | 2022-08-25 | President And Fellows Of Harvard College | Methods and compositions for gene delivery |
| CA3174500A1 (en) * | 2020-04-01 | 2021-10-07 | University Of Florida Research Foundation, Incorporated | Dual aav-myo7a vectors with improved safety for the treatment of ush1b |
| WO2022036318A1 (en) * | 2020-08-14 | 2022-02-17 | Case Western Reserve University | Plasmid vectors and nanoparticles for treating ocular disorders |
| CA3218195A1 (en) * | 2021-05-07 | 2022-11-10 | Robin Ali | Abca4 genome editing |
| US20240425876A1 (en) * | 2022-02-25 | 2024-12-26 | Chigenovo Co., Ltd. | Expression cassette combinations and uses thereof |
| WO2024257061A1 (en) * | 2023-06-16 | 2024-12-19 | Arkasubhra Ghosh | A hybrid dual aav vector system with splice enhancer elements for expression of large genes |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101287837B (zh) * | 2005-10-20 | 2015-03-18 | 尤尼克尔生物制药股份有限公司 | 昆虫细胞中生产的改进的aav载体 |
| US20090214478A1 (en) * | 2008-02-21 | 2009-08-27 | Alberto Auricchio | Method of treating ocular diseases by gene therapy |
| BR112012002628B1 (pt) * | 2009-08-07 | 2021-09-08 | Transgene Sa | Composição imunogênica, molécula de ácido nucleico, vetor, partícula viral infecciosa, micro-organismo transgênico, composição, usos da composição imunogênica e kit de partes |
| WO2013075008A1 (en) * | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| DK2986635T3 (en) * | 2013-04-18 | 2019-01-28 | Fond Telethon | EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS |
-
2017
- 2017-06-14 EP EP17732175.9A patent/EP3472328A1/en not_active Withdrawn
- 2017-06-14 WO PCT/GB2017/051741 patent/WO2017216560A1/en not_active Ceased
- 2017-06-14 SG SG11201811244SA patent/SG11201811244SA/en unknown
- 2017-06-14 CA CA3025445A patent/CA3025445A1/en not_active Abandoned
- 2017-06-14 MX MX2018015629A patent/MX2018015629A/es unknown
- 2017-06-14 RU RU2019100525A patent/RU2765826C2/ru active
- 2017-06-14 US US16/309,428 patent/US20190309326A1/en not_active Abandoned
- 2017-06-14 KR KR1020197001281A patent/KR20190020745A/ko not_active Ceased
- 2017-06-14 AU AU2017286623A patent/AU2017286623A1/en not_active Abandoned
- 2017-06-14 CN CN201780037639.9A patent/CN109642242A/zh active Pending
- 2017-06-14 BR BR112018075855-8A patent/BR112018075855A2/pt not_active Application Discontinuation
- 2017-06-14 JP JP2018565324A patent/JP2019523648A/ja active Pending
-
2018
- 2018-12-05 IL IL263523A patent/IL263523A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CN109642242A (zh) | 2019-04-16 |
| SG11201811244SA (en) | 2019-01-30 |
| KR20190020745A (ko) | 2019-03-04 |
| RU2019100525A (ru) | 2020-07-15 |
| AU2017286623A1 (en) | 2018-12-20 |
| US20190309326A1 (en) | 2019-10-10 |
| RU2765826C2 (ru) | 2022-02-03 |
| IL263523A (en) | 2019-01-31 |
| WO2017216560A1 (en) | 2017-12-21 |
| MX2018015629A (es) | 2019-09-26 |
| BR112018075855A2 (pt) | 2019-04-02 |
| CA3025445A1 (en) | 2017-12-21 |
| JP2019523648A (ja) | 2019-08-29 |
| EP3472328A1 (en) | 2019-04-24 |