KR20230043794A - 변이체 캡시드를 갖는 아데노-연관 바이러스 비리온 및 이의 사용 방법 - Google Patents
변이체 캡시드를 갖는 아데노-연관 바이러스 비리온 및 이의 사용 방법 Download PDFInfo
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- KR20230043794A KR20230043794A KR1020227045801A KR20227045801A KR20230043794A KR 20230043794 A KR20230043794 A KR 20230043794A KR 1020227045801 A KR1020227045801 A KR 1020227045801A KR 20227045801 A KR20227045801 A KR 20227045801A KR 20230043794 A KR20230043794 A KR 20230043794A
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- 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
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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
- A61K48/005—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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
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- 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
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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- 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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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
- 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
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14145—Special targeting system for viral vectors
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Ophthalmology & Optometry (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Epidemiology (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063032206P | 2020-05-29 | 2020-05-29 | |
| US63/032,206 | 2020-05-29 | ||
| US202163187154P | 2021-05-11 | 2021-05-11 | |
| US63/187,154 | 2021-05-11 | ||
| PCT/US2021/034624 WO2021243085A2 (en) | 2020-05-29 | 2021-05-27 | Adeno-associated virus virions with variant capsids and methods of use thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20230043794A true KR20230043794A (ko) | 2023-03-31 |
Family
ID=78706867
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020227045801A Pending KR20230043794A (ko) | 2020-05-29 | 2021-05-27 | 변이체 캡시드를 갖는 아데노-연관 바이러스 비리온 및 이의 사용 방법 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US12031147B2 (https=) |
| EP (1) | EP4157299A4 (https=) |
| JP (1) | JP2023528801A (https=) |
| KR (1) | KR20230043794A (https=) |
| CN (1) | CN116249772A (https=) |
| AU (1) | AU2021280317A1 (https=) |
| CA (1) | CA3179597A1 (https=) |
| MX (1) | MX2022015048A (https=) |
| WO (1) | WO2021243085A2 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2022150776A1 (en) | 2021-01-11 | 2022-07-14 | Vedere Bio Ii, Inc. | OPTOGENETIC COMPOSITIONS COMPRISING A CBh PROMOTER SEQUENCE AND METHODS FOR USE |
| MX2024004217A (es) | 2021-10-08 | 2024-06-26 | Dyno Therapeutics Inc | Variantes de cápside y métodos de uso de estas. |
| TW202502803A (zh) | 2023-03-10 | 2025-01-16 | 美商戴諾治療公司 | 衣殼多肽及其使用方法 |
| CN118791624B (zh) * | 2023-04-11 | 2026-04-03 | 苏州星明优健生物技术有限公司 | 用于治疗视网膜神经退行性疾病的新型gpcr嵌合视蛋白 |
| TW202525832A (zh) | 2023-08-31 | 2025-07-01 | 美商戴諾治療公司 | 衣殼多肽及其使用方法(二) |
| TW202545971A (zh) | 2024-02-08 | 2025-12-01 | 美商戴諾治療公司 | 殼體多肽及其使用方法 |
| WO2026064442A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| CN118955654B (zh) * | 2024-10-15 | 2025-05-16 | 朗信启昇(苏州)生物制药有限公司 | 具有变异衣壳的腺相关病毒病毒体及其用途 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8663624B2 (en) * | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| HRP20171334T1 (hr) | 2011-04-22 | 2017-11-17 | The Regents Of The University Of California | Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe |
| MY187898A (en) * | 2015-03-02 | 2021-10-27 | Adverum Biotechnologies Inc | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| JP6836999B2 (ja) | 2015-03-24 | 2021-03-03 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California | アデノ随伴ウイルス変異体及びその使用方法 |
| FI3445773T3 (fi) * | 2016-05-13 | 2023-03-30 | 4D Molecular Therapeutics Inc | Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä |
| EP3827812B1 (en) | 2016-07-29 | 2025-10-29 | The Regents of the University of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| CA3040179A1 (en) * | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| WO2018134168A1 (en) * | 2017-01-17 | 2018-07-26 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods of expressing a polynucleotide of interest in the cone photoreceptors |
| WO2019006182A1 (en) | 2017-06-30 | 2019-01-03 | The Regents Of The University Of California | VIRIONS OF ADENO-ASSOCIATED VIRUSES OF CAPSID VARIANTS AND METHODS OF USE THEREOF |
| JP2020534788A (ja) * | 2017-08-28 | 2020-12-03 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | アデノ随伴ウイルスカプシド変異体及びその使用方法 |
| UY39772A (es) * | 2021-05-21 | 2023-01-31 | Novartis Ag | Composiciones y métodos para mejorar la función visual |
-
2021
- 2021-05-27 JP JP2022573159A patent/JP2023528801A/ja active Pending
- 2021-05-27 WO PCT/US2021/034624 patent/WO2021243085A2/en not_active Ceased
- 2021-05-27 US US17/332,855 patent/US12031147B2/en active Active
- 2021-05-27 CA CA3179597A patent/CA3179597A1/en active Pending
- 2021-05-27 EP EP21812280.2A patent/EP4157299A4/en not_active Withdrawn
- 2021-05-27 CN CN202180041153.9A patent/CN116249772A/zh active Pending
- 2021-05-27 KR KR1020227045801A patent/KR20230043794A/ko active Pending
- 2021-05-27 AU AU2021280317A patent/AU2021280317A1/en not_active Abandoned
- 2021-05-27 MX MX2022015048A patent/MX2022015048A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| US20210371879A1 (en) | 2021-12-02 |
| CN116249772A (zh) | 2023-06-09 |
| CA3179597A1 (en) | 2021-12-02 |
| AU2021280317A1 (en) | 2023-01-05 |
| WO2021243085A3 (en) | 2022-01-06 |
| EP4157299A2 (en) | 2023-04-05 |
| EP4157299A4 (en) | 2024-10-02 |
| JP2023528801A (ja) | 2023-07-06 |
| WO2021243085A2 (en) | 2021-12-02 |
| US12031147B2 (en) | 2024-07-09 |
| MX2022015048A (es) | 2023-03-08 |
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| US12031147B2 (en) | Adeno-associated virus virions with variant capsids and methods of use thereof | |
| HK40051378A (en) | Adeno-associated virus virions with variant capsid and methods of use thereof |
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