CN114127090A - 在大脑中具有增强的特异性的病毒组合物 - Google Patents
在大脑中具有增强的特异性的病毒组合物 Download PDFInfo
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- CN114127090A CN114127090A CN202080043549.2A CN202080043549A CN114127090A CN 114127090 A CN114127090 A CN 114127090A CN 202080043549 A CN202080043549 A CN 202080043549A CN 114127090 A CN114127090 A CN 114127090A
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- 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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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- 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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- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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- 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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- 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
- C12N2750/14151—Methods of production or purification of viral material
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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
- C12N2750/14171—Demonstrated in vivo effect
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- Molecular Biology (AREA)
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- Biophysics (AREA)
- Gastroenterology & Hepatology (AREA)
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- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962832826P | 2019-04-11 | 2019-04-11 | |
| US62/832,826 | 2019-04-11 | ||
| PCT/US2020/027708 WO2020210655A1 (en) | 2019-04-11 | 2020-04-10 | Virus compositions with enhanced specificity in the brain |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN114127090A true CN114127090A (zh) | 2022-03-01 |
Family
ID=72751358
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202080043549.2A Pending CN114127090A (zh) | 2019-04-11 | 2020-04-10 | 在大脑中具有增强的特异性的病毒组合物 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20220220502A1 (https=) |
| EP (1) | EP3953371A4 (https=) |
| JP (2) | JP2022526018A (https=) |
| KR (1) | KR20220007056A (https=) |
| CN (1) | CN114127090A (https=) |
| AU (1) | AU2020271108A1 (https=) |
| CA (1) | CA3136658A1 (https=) |
| SG (1) | SG11202111249YA (https=) |
| WO (1) | WO2020210655A1 (https=) |
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN116693633A (zh) * | 2023-02-21 | 2023-09-05 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN116983437A (zh) * | 2023-07-26 | 2023-11-03 | 武汉大学中南医院 | 一种αOlig2的无损影像标记方法和产物及其用途 |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020014471A1 (en) | 2018-07-11 | 2020-01-16 | The Brigham And Women's Hospital, Inc. | Methods and compositions for delivery of agents across the blood-brain barrier |
| CN113966399A (zh) | 2018-09-26 | 2022-01-21 | 加州理工学院 | 用于靶向基因疗法的腺相关病毒组合物 |
| CN113166208B (zh) | 2018-10-02 | 2024-11-22 | 沃雅戈治疗公司 | 重新定向aav衣壳的嗜性 |
| EP4061947A4 (en) | 2019-11-22 | 2024-04-24 | California Institute of Technology | METHODS FOR ROBUST CONTROL OF GENE EXPRESSION |
| CN115279400B (zh) | 2020-01-10 | 2026-04-28 | 布里格姆妇女医院 | 用于跨血脑屏障递送免疫治疗剂以治疗脑癌的方法和组合物 |
| TWI900553B (zh) | 2020-05-13 | 2025-10-11 | 美商航海家醫療公司 | Aav蛋白殼之趨性重定向 |
| EP4263575A4 (en) * | 2020-12-16 | 2025-05-07 | Children's Medical Research Institute | Adeno-associated virus capsids and vectors |
| CN116917491A (zh) * | 2020-12-18 | 2023-10-20 | Ac免疫有限公司 | 抗体递送 |
| US20250281643A1 (en) * | 2021-04-13 | 2025-09-11 | Capsida, Inc. | Selected aav compositions having preferred brain enrichment |
| JP2024513949A (ja) * | 2021-04-13 | 2024-03-27 | カプシダ, インコーポレイテッド | 脳内発現レベルが高いaav組成物 |
| WO2022221421A2 (en) * | 2021-04-13 | 2022-10-20 | Capsida, Inc. | Aav compositions with high brain expression for treating mucopolysaccharidosis ii |
| AU2022266615A1 (en) * | 2021-04-27 | 2023-11-09 | The General Hospital Corporation | Aav capsids and uses thereof |
| EP4334332A4 (en) * | 2021-05-04 | 2025-11-12 | California Inst Of Techn | VAA RECOMMENDANTS FOR ADMINISTRATION TO THE CENTRAL NERVOUS SYSTEM AND CEREBRAL VASCULATURE |
| US20240325568A1 (en) * | 2021-07-20 | 2024-10-03 | The Broad Institute, Inc. | Engineered targeting compositions for endothelial cells of the central nervous system vasculature and methods of use thereof |
| KR20240095539A (ko) | 2021-10-08 | 2024-06-25 | 디노 테라퓨틱스, 인코포레이티드 | 캡시드 변이체 및 이의 사용 방법 |
| EP4426716A1 (en) | 2021-11-02 | 2024-09-11 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| WO2023091934A1 (en) * | 2021-11-16 | 2023-05-25 | California Institute Of Technology | Adeno-associated virus compositions having preferred heart and skeletal muscle enrichment |
| US20250297280A2 (en) * | 2022-02-01 | 2025-09-25 | The Broad Institute, Inc. | Adeno-associated viral vectors and uses thereof |
| WO2023150632A2 (en) * | 2022-02-02 | 2023-08-10 | The Broad Institute, Inc. | Targeting moieties promoting transduction of central nervous system cells and tissues and methods of use |
| EP4532515A1 (en) * | 2022-06-02 | 2025-04-09 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| KR20240014846A (ko) * | 2022-07-26 | 2024-02-02 | 숙명여자대학교산학협력단 | Aav2-anks1a 재조합 바이러스를 포함하는 알츠하이머 질병 치료용 조성물 |
| WO2024086747A1 (en) | 2022-10-19 | 2024-04-25 | Affinia Therapeutics Inc. | Recombinant aavs with improved tropism and specificity |
| WO2024191778A1 (en) | 2023-03-10 | 2024-09-19 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| AU2024332181A1 (en) | 2023-08-31 | 2026-02-12 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2025082176A1 (zh) * | 2023-10-19 | 2025-04-24 | 北京脑科学与类脑研究所 | 一种脑血管内皮细胞嵌合型启动子和重组表达载体及应用 |
| WO2025155923A1 (en) * | 2024-01-17 | 2025-07-24 | The Broad Institute, Inc. | Aav capsid modifications that enable improved cns-wide gene delivery through interactions with the transferrin receptor |
| 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 |
| WO2025250454A1 (en) | 2024-05-28 | 2025-12-04 | University Of Rochester | Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo |
| WO2026041788A1 (en) * | 2024-08-22 | 2026-02-26 | Uniqure Biopharma B.V | Novel bbb interacting capsids |
| WO2026064442A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20150079038A1 (en) * | 2013-09-13 | 2015-03-19 | California Institute Of Technology | Selective recovery |
| CN104797593A (zh) * | 2012-09-28 | 2015-07-22 | 北卡罗来纳-查佩尔山大学 | 靶向少突胶质细胞的aav载体 |
| WO2017100671A1 (en) * | 2015-12-11 | 2017-06-15 | California Institute Of Technology | TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs) |
Family Cites Families (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6947739B2 (ja) * | 2016-02-16 | 2021-10-13 | ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー | 既存のヒト中和抗体に対して耐性である新規組換えアデノ随伴ウイルスカプシド |
| 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 |
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2020
- 2020-04-10 JP JP2021559829A patent/JP2022526018A/ja not_active Withdrawn
- 2020-04-10 SG SG11202111249YA patent/SG11202111249YA/en unknown
- 2020-04-10 WO PCT/US2020/027708 patent/WO2020210655A1/en not_active Ceased
- 2020-04-10 AU AU2020271108A patent/AU2020271108A1/en active Pending
- 2020-04-10 US US17/602,505 patent/US20220220502A1/en active Pending
- 2020-04-10 CA CA3136658A patent/CA3136658A1/en active Pending
- 2020-04-10 CN CN202080043549.2A patent/CN114127090A/zh active Pending
- 2020-04-10 EP EP20787580.8A patent/EP3953371A4/en active Pending
- 2020-04-10 KR KR1020217036564A patent/KR20220007056A/ko active Pending
-
2025
- 2025-04-11 JP JP2025065562A patent/JP2025106514A/ja active Pending
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN104797593A (zh) * | 2012-09-28 | 2015-07-22 | 北卡罗来纳-查佩尔山大学 | 靶向少突胶质细胞的aav载体 |
| US20150079038A1 (en) * | 2013-09-13 | 2015-03-19 | California Institute Of Technology | Selective recovery |
| US20180230186A1 (en) * | 2013-09-13 | 2018-08-16 | California Institute Of Technology | Selective recovery |
| WO2017100671A1 (en) * | 2015-12-11 | 2017-06-15 | California Institute Of Technology | TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs) |
Non-Patent Citations (3)
| Title |
|---|
| JAKOB KÖRBELIN等: "A brain microvasculature endothelial cell‐specific viral vector with the potential to treat neurovascular and neurological diseases", EMBO MOL MED, vol. 8, no. 6, 30 June 2016 (2016-06-30), pages 609 - 625 * |
| KEN Y CHAN等: "Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems", NATURE NEUROSCIENCE, vol. 20, no. 8, 26 June 2017 (2017-06-26), pages 1172 - 1179, XP055527909, DOI: 10.1038/nn.4593 * |
| 殷子斐等: "提高重组型腺相关病毒转导效率的研究现状", 生物技术通报, vol. 31, no. 9, 31 December 2015 (2015-12-31), pages 49 - 59 * |
Cited By (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN116693633A (zh) * | 2023-02-21 | 2023-09-05 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN116693633B (zh) * | 2023-02-21 | 2023-12-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN116983437A (zh) * | 2023-07-26 | 2023-11-03 | 武汉大学中南医院 | 一种αOlig2的无损影像标记方法和产物及其用途 |
Also Published As
| Publication number | Publication date |
|---|---|
| JP2022526018A (ja) | 2022-05-20 |
| SG11202111249YA (en) | 2021-11-29 |
| AU2020271108A1 (en) | 2021-11-11 |
| JP2025106514A (ja) | 2025-07-15 |
| EP3953371A4 (en) | 2023-04-12 |
| WO2020210655A1 (en) | 2020-10-15 |
| US20220220502A1 (en) | 2022-07-14 |
| KR20220007056A (ko) | 2022-01-18 |
| CA3136658A1 (en) | 2020-10-15 |
| EP3953371A1 (en) | 2022-02-16 |
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