CN117321213A - 具有优选表达水平的腺相关病毒组合物 - Google Patents

具有优选表达水平的腺相关病毒组合物 Download PDF

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Publication number
CN117321213A
CN117321213A CN202180071840.5A CN202180071840A CN117321213A CN 117321213 A CN117321213 A CN 117321213A CN 202180071840 A CN202180071840 A CN 202180071840A CN 117321213 A CN117321213 A CN 117321213A
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CN
China
Prior art keywords
seq
amino acid
further provided
acid selected
aav capsid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CN202180071840.5A
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English (en)
Chinese (zh)
Inventor
维维安娜·格拉迪纳鲁
N·C·弗利扎尼
N·S·戈登
K·博伊特纳
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Cassida Co
California Institute of Technology
Original Assignee
Cassida Co
California Institute of Technology
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Filing date
Publication date
Application filed by Cassida Co, California Institute of Technology filed Critical Cassida Co
Publication of CN117321213A publication Critical patent/CN117321213A/zh
Pending legal-status Critical Current

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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
    • 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
    • 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

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202180071840.5A 2020-08-21 2021-08-20 具有优选表达水平的腺相关病毒组合物 Pending CN117321213A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063068614P 2020-08-21 2020-08-21
US63/068,614 2020-08-21
PCT/US2021/046904 WO2022040527A2 (en) 2020-08-21 2021-08-20 Adeno-associated virus compositions having preferred expression levels

Publications (1)

Publication Number Publication Date
CN117321213A true CN117321213A (zh) 2023-12-29

Family

ID=80323313

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180071840.5A Pending CN117321213A (zh) 2020-08-21 2021-08-20 具有优选表达水平的腺相关病毒组合物

Country Status (7)

Country Link
US (1) US20230303630A1 (https=)
EP (1) EP4199972A4 (https=)
JP (1) JP2023538129A (https=)
CN (1) CN117321213A (https=)
AU (1) AU2021329527A1 (https=)
CA (1) CA3192600A1 (https=)
WO (1) WO2022040527A2 (https=)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
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
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
CA3254573A1 (en) * 2022-03-24 2023-09-28 Capsida, Inc. SELECTED VAA COMPOSITIONS WITH A PREFERRED BRAIN ENRICHEMENT
CN114774373B (zh) * 2022-04-27 2024-07-05 北京市农林科学院 信鸽新城疫病毒基因工程改造弱毒株及其制备方法和应用
EP4269426A1 (en) * 2022-04-29 2023-11-01 Ospedale San Raffaele S.r.l. Gene therapy
EP4519284A2 (en) * 2022-05-06 2025-03-12 Capsida, Inc. Adeno-associated virus compositions having increased brain enrichment
CA3256915A1 (en) * 2022-05-17 2023-11-23 Capsida, Inc. SELECTED ADENE-ASSOCIATED VIRUS COMPOSITIONS HAVING PREFERRED LEVELS OF CEREBRAL, SPINAL CORD AND/OR CARDIAC EXPRESSION
US20250346637A1 (en) * 2022-06-16 2025-11-13 Capsida, Inc. Adeno-associated virus compositions having increased brain enrichment and decreased liver enrichment
WO2024086747A1 (en) 2022-10-19 2024-04-25 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
EP4605538A2 (en) * 2022-10-19 2025-08-27 Capsida Biotherapeutics, Inc. Adeno-associated virus compositions having preferredbrain enrichment and low liver enrichment
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2024199270A1 (zh) * 2023-03-27 2024-10-03 浙江大学 氨基酸序列的生成与筛选方法及其系统
KR20260045798A (ko) * 2023-06-30 2026-04-03 캡시다 바이오테라퓨틱스, 인크. 증가된 뇌 풍부화를 갖는 아데노-연관 바이러스 조성물
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
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
WO2025213066A1 (en) * 2024-04-05 2025-10-09 Capsida Biotherapeutics, Inc. Adeno-associated virus compositions for treating parkinson's disease (pd)
WO2025231383A1 (en) * 2024-05-03 2025-11-06 Capsida Biotherapeutics, Inc. Compositions and methods of delivering a therapeutic across the blood-brain barrier via the pl16 receptor
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6710036B2 (en) * 1997-07-25 2004-03-23 Avigen, Inc. Induction of immune response to antigens expressed by recombinant adeno-associated virus
CN105755044A (zh) * 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
DE102014207498A1 (de) * 2014-04-17 2015-10-22 Universitätsklinikum Hamburg-Eppendorf Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark
KR102423442B1 (ko) * 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
AU2017302013B2 (en) * 2016-07-29 2022-05-26 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
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
WO2020028751A2 (en) * 2018-08-03 2020-02-06 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
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
EP3856762A1 (en) * 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
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

Also Published As

Publication number Publication date
WO2022040527A2 (en) 2022-02-24
US20230303630A1 (en) 2023-09-28
EP4199972A2 (en) 2023-06-28
AU2021329527A1 (en) 2023-03-23
CA3192600A1 (en) 2022-02-24
JP2023538129A (ja) 2023-09-06
WO2022040527A3 (en) 2022-05-05
EP4199972A4 (en) 2025-01-15

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