KR20220007056A - 뇌에서 증진된 특이성을 갖는 바이러스 조성물 - Google Patents
뇌에서 증진된 특이성을 갖는 바이러스 조성물 Download PDFInfo
- Publication number
- KR20220007056A KR20220007056A KR1020217036564A KR20217036564A KR20220007056A KR 20220007056 A KR20220007056 A KR 20220007056A KR 1020217036564 A KR1020217036564 A KR 1020217036564A KR 20217036564 A KR20217036564 A KR 20217036564A KR 20220007056 A KR20220007056 A KR 20220007056A
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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
- 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
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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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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- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
- C12N2750/14122—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
- C12N2750/14151—Methods of production or purification of viral material
-
- 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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- 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)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Virology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- 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 |
|---|---|
| KR20220007056A true KR20220007056A (ko) | 2022-01-18 |
Family
ID=72751358
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020217036564A Pending KR20220007056A (ko) | 2019-04-11 | 2020-04-10 | 뇌에서 증진된 특이성을 갖는 바이러스 조성물 |
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=) |
Families Citing this family (32)
| 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 |
| 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 |
| WO2024191778A1 (en) | 2023-03-10 | 2024-09-19 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| CN116983437B (zh) * | 2023-07-26 | 2025-08-12 | 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 |
| 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 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2900686B1 (en) * | 2012-09-28 | 2020-06-10 | The University of North Carolina At Chapel Hill | Aav vectors targeted to oligodendrocytes |
| ES2739288T3 (es) * | 2013-09-13 | 2020-01-30 | California Inst Of Techn | Recuperación selectiva |
| KR102423442B1 (ko) * | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| 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 |
-
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
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 |
| CN114127090A (zh) | 2022-03-01 |
| US20220220502A1 (en) | 2022-07-14 |
| CA3136658A1 (en) | 2020-10-15 |
| EP3953371A1 (en) | 2022-02-16 |
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