JP2023538129A - 好ましい発現レベルを有するアデノ随伴ウイルス組成物 - Google Patents

好ましい発現レベルを有するアデノ随伴ウイルス組成物 Download PDF

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Publication number
JP2023538129A
JP2023538129A JP2023512697A JP2023512697A JP2023538129A JP 2023538129 A JP2023538129 A JP 2023538129A JP 2023512697 A JP2023512697 A JP 2023512697A JP 2023512697 A JP2023512697 A JP 2023512697A JP 2023538129 A JP2023538129 A JP 2023538129A
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Japan
Prior art keywords
seq
amino acid
acid selected
aav capsid
further provided
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JP2023512697A
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English (en)
Japanese (ja)
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JP2023538129A5 (https=
Inventor
ヴィヴィアナ グラディナル，
ニコラス シー． フリトザニス，
ニコラス エス． ゴーデン，
カール ビュートナー，
Original Assignee
カプシダ， インコーポレイテッド
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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Publication of JP2023538129A publication Critical patent/JP2023538129A/ja
Publication of JP2023538129A5 publication Critical patent/JP2023538129A5/ja
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)
JP2023512697A 2020-08-21 2021-08-20 好ましい発現レベルを有するアデノ随伴ウイルス組成物 Pending JP2023538129A (ja)

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 (2)

Publication Number Publication Date
JP2023538129A true JP2023538129A (ja) 2023-09-06
JP2023538129A5 JP2023538129A5 (https=) 2024-08-28

Family

ID=80323313

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023512697A Pending JP2023538129A (ja) 2020-08-21 2021-08-20 好ましい発現レベルを有するアデノ随伴ウイルス組成物

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=)

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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

Citations (5)

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Publication number Priority date Publication date Assignee Title
JP2017513486A (ja) * 2014-04-17 2017-06-01 ウニヴェルズィテーツクリニクム ハンブルク−エッペンドルフＵｎｉｖｅｒｓｉｔａｅｔｓｋｌｉｎｉｋｕｍ Ｈａｍｂｕｒｇ−Ｅｐｐｅｎｄｏｒｆ 脳および脊髄に標的化遺伝子移入するためのウイルスベクター
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
WO2020028751A2 (en) * 2018-08-03 2020-02-06 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
WO2020068990A1 (en) * 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
WO2020072683A1 (en) * 2018-10-02 2020-04-09 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids

Family Cites Families (5)

* Cited by examiner, † Cited by third party
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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
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
EP3856762A1 (en) * 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017513486A (ja) * 2014-04-17 2017-06-01 ウニヴェルズィテーツクリニクム ハンブルク−エッペンドルフＵｎｉｖｅｒｓｉｔａｅｔｓｋｌｉｎｉｋｕｍ Ｈａｍｂｕｒｇ−Ｅｐｐｅｎｄｏｒｆ 脳および脊髄に標的化遺伝子移入するためのウイルスベクター
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
WO2020028751A2 (en) * 2018-08-03 2020-02-06 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
WO2020068990A1 (en) * 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
WO2020072683A1 (en) * 2018-10-02 2020-04-09 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids

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
CN117321213A (zh) 2023-12-29
WO2022040527A3 (en) 2022-05-05
EP4199972A4 (en) 2025-01-15

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