CN117157309A - 衣壳变异的重组腺相关病毒及其应用 - Google Patents
衣壳变异的重组腺相关病毒及其应用 Download PDFInfo
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- CN117157309A CN117157309A CN202280028081.9A CN202280028081A CN117157309A CN 117157309 A CN117157309 A CN 117157309A CN 202280028081 A CN202280028081 A CN 202280028081A CN 117157309 A CN117157309 A CN 117157309A
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- Prior art keywords
- seq
- aav
- cells
- capsid protein
- raav
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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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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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
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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
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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
- 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
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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
- A61K9/0048—Eye, e.g. artificial tears
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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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- 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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- 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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- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- 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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- 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
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Molecular Biology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Biophysics (AREA)
- Ophthalmology & Optometry (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Gastroenterology & Hepatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Dermatology (AREA)
- Immunology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN202110594986 | 2021-05-28 | ||
| CN202110594986X | 2021-05-28 | ||
| PCT/CN2022/095422 WO2022247917A1 (zh) | 2021-05-28 | 2022-05-27 | 衣壳变异的重组腺相关病毒及其应用 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN117157309A true CN117157309A (zh) | 2023-12-01 |
Family
ID=84228446
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202280028081.9A Pending CN117157309A (zh) | 2021-05-28 | 2022-05-27 | 衣壳变异的重组腺相关病毒及其应用 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20240252679A1 (https=) |
| EP (1) | EP4349852A4 (https=) |
| JP (1) | JP2024519888A (https=) |
| KR (1) | KR20240014477A (https=) |
| CN (1) | CN117157309A (https=) |
| AU (1) | AU2022281825A1 (https=) |
| BR (1) | BR112023024375A2 (https=) |
| CA (1) | CA3219898A1 (https=) |
| MX (1) | MX2023014041A (https=) |
| TW (1) | TW202313096A (https=) |
| WO (1) | WO2022247917A1 (https=) |
Cited By (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN118754952A (zh) * | 2024-09-06 | 2024-10-11 | 上海玮美基因科技有限责任公司 | 一种新型腺相关病毒及其应用 |
| CN119039401A (zh) * | 2024-08-19 | 2024-11-29 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN120118955A (zh) * | 2025-03-05 | 2025-06-10 | 广州派真生物技术有限公司 | 一种高产aav的包装质粒及应用 |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7842082B2 (ja) * | 2020-07-21 | 2026-04-07 | エフティージェン コーポレーション | 眼疾患を処置するための組成物および方法 |
| CN116789739B (zh) * | 2022-12-08 | 2024-01-26 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN118307648A (zh) * | 2023-01-09 | 2024-07-09 | 广东克冠达医药科技有限公司 | 腺相关病毒衣壳蛋白衍生多肽及其用途 |
| CN118359687A (zh) * | 2023-01-18 | 2024-07-19 | 上海朗昇生物科技有限公司 | 一种新衣壳蛋白变体以及其用途 |
| CN118420721A (zh) * | 2023-01-31 | 2024-08-02 | 上海朗昇生物科技有限公司 | 一种组织嗜性的腺相关病毒衣壳及其用途 |
| EP4688807A1 (en) * | 2023-03-31 | 2026-02-11 | Exegenesis Bio Singapore Pte. Ltd. | Variant aav9 capsid proteins and uses thereof |
| AU2024276934A1 (en) * | 2023-05-23 | 2025-12-18 | Innovec Biotherapeutics | Adeno-associated virus capsid protein, adeno-associated virus containing same, vector and use thereof |
| CN117801076B (zh) * | 2023-06-09 | 2024-05-31 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN116836237B (zh) * | 2023-06-16 | 2023-12-05 | 广州译码基因科技有限公司 | 提高视网膜靶向性的aav病毒衣壳蛋白突变体及其应用 |
| TW202508610A (zh) | 2023-08-23 | 2025-03-01 | 大陸商上海瑞宏迪醫藥有限公司 | 醫藥組成物及其用途 |
| CN119569833A (zh) * | 2023-09-06 | 2025-03-07 | 北京锦篮基因科技有限公司 | 靶向骨骼肌的aav衣壳蛋白变体及其用途 |
| CN119569834A (zh) * | 2023-09-06 | 2025-03-07 | 北京锦篮基因科技有限公司 | 靶向心肌细胞的aav衣壳蛋白变体及其用途 |
| CN120230798A (zh) * | 2023-12-29 | 2025-07-01 | 上海朗昇生物科技有限公司 | 用于视网膜基因递送的重组腺相关病毒载体及其应用 |
| WO2026033140A1 (en) * | 2024-08-09 | 2026-02-12 | Revvity Gene Delivery Gmbh | Variant aav capsid polypeptides targeting the eye |
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| CA3040179A1 (en) * | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| JP2020510424A (ja) | 2017-02-28 | 2020-04-09 | アドヴェラム バイオテクノロジーズ, インコーポレイテッド | 改変aavカプシドおよびその使用 |
| CN111770999A (zh) * | 2017-11-27 | 2020-10-13 | 4D分子治疗有限公司 | 腺相关病毒变体衣壳和用于抑制血管生成的应用 |
| HRP20260137T1 (hr) * | 2019-09-11 | 2026-03-13 | Adverum Biotechnologies, Inc. | Liječenje očnih neovaskularnih bolesti upotrebom aav2 varijanti koje kodiraju aflibercept |
| CN114729009A (zh) * | 2019-10-31 | 2022-07-08 | 伯尔尼大学 | 用于眼部基因递送的aav载体变体 |
-
2022
- 2022-05-27 US US18/564,584 patent/US20240252679A1/en active Pending
- 2022-05-27 MX MX2023014041A patent/MX2023014041A/es unknown
- 2022-05-27 EP EP22810641.5A patent/EP4349852A4/en active Pending
- 2022-05-27 JP JP2023571788A patent/JP2024519888A/ja active Pending
- 2022-05-27 CN CN202280028081.9A patent/CN117157309A/zh active Pending
- 2022-05-27 TW TW111119884A patent/TW202313096A/zh unknown
- 2022-05-27 AU AU2022281825A patent/AU2022281825A1/en active Pending
- 2022-05-27 WO PCT/CN2022/095422 patent/WO2022247917A1/zh not_active Ceased
- 2022-05-27 BR BR112023024375A patent/BR112023024375A2/pt unknown
- 2022-05-27 KR KR1020237043802A patent/KR20240014477A/ko active Pending
- 2022-05-27 CA CA3219898A patent/CA3219898A1/en active Pending
Cited By (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN119039401A (zh) * | 2024-08-19 | 2024-11-29 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2026040404A1 (zh) * | 2024-08-19 | 2026-02-26 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN118754952A (zh) * | 2024-09-06 | 2024-10-11 | 上海玮美基因科技有限责任公司 | 一种新型腺相关病毒及其应用 |
| CN120118955A (zh) * | 2025-03-05 | 2025-06-10 | 广州派真生物技术有限公司 | 一种高产aav的包装质粒及应用 |
Also Published As
| Publication number | Publication date |
|---|---|
| BR112023024375A2 (pt) | 2024-02-15 |
| WO2022247917A1 (zh) | 2022-12-01 |
| CA3219898A1 (en) | 2023-11-21 |
| EP4349852A1 (en) | 2024-04-10 |
| MX2023014041A (es) | 2023-12-15 |
| JP2024519888A (ja) | 2024-05-21 |
| EP4349852A4 (en) | 2025-09-03 |
| KR20240014477A (ko) | 2024-02-01 |
| AU2022281825A1 (en) | 2024-01-18 |
| US20240252679A1 (en) | 2024-08-01 |
| TW202313096A (zh) | 2023-04-01 |
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