JP2022526018A - 脳内への特異性が増強されたウイルス組成物 - Google Patents

脳内への特異性が増強されたウイルス組成物 Download PDF

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JP2022526018A
JP2022526018A JP2021559829A JP2021559829A JP2022526018A JP 2022526018 A JP2022526018 A JP 2022526018A JP 2021559829 A JP2021559829 A JP 2021559829A JP 2021559829 A JP2021559829 A JP 2021559829A JP 2022526018 A JP2022526018 A JP 2022526018A
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aav capsid
aav
amino acid
cells
cases
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ヴィヴィアナ グラディナル，
スリプリヤ ラヴィンドラ クマール，
ベンジャミン デバーマン，
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カリフォルニア インスティテュート オブ テクノロジー
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2021559829A 2019-04-11 2020-04-10 脳内への特異性が増強されたウイルス組成物 Withdrawn JP2022526018A (ja)

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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

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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)

* Cited by examiner, † Cited by third party
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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019506141A (ja) * 2015-12-11 2019-03-07 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス（ａａｖ）を誘導するための標的指向性ペプチド
JP2019509032A (ja) * 2016-02-16 2019-04-04 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

Family Cites Families (3)

* Cited by examiner, † Cited by third party
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
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

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019506141A (ja) * 2015-12-11 2019-03-07 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス（ａａｖ）を誘導するための標的指向性ペプチド
JP2019509032A (ja) * 2016-02-16 2019-04-04 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

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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
KR20220007056A (ko) 2022-01-18
CA3136658A1 (en) 2020-10-15
EP3953371A1 (en) 2022-02-16

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