JP2023513004A5 - - Google Patents

Info

Publication number
JP2023513004A5
JP2023513004A5 JP2022545976A JP2022545976A JP2023513004A5 JP 2023513004 A5 JP2023513004 A5 JP 2023513004A5 JP 2022545976 A JP2022545976 A JP 2022545976A JP 2022545976 A JP2022545976 A JP 2022545976A JP 2023513004 A5 JP2023513004 A5 JP 2023513004A5
Authority
JP
Japan
Prior art keywords
amino acid
sequence
capsid protein
raav
nucleic acid
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
JP2022545976A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023513004A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/015695 external-priority patent/WO2021155137A1/en
Publication of JP2023513004A publication Critical patent/JP2023513004A/ja
Publication of JP2023513004A5 publication Critical patent/JP2023513004A5/ja
Pending legal-status Critical Current

Links

JP2022545976A 2020-01-29 2021-01-29 眼科遺伝子療法のための改変されたアデノ随伴ウイルスカプシドタンパク質およびその使用方法 Pending JP2023513004A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062967416P 2020-01-29 2020-01-29
US62/967,416 2020-01-29
PCT/US2021/015695 WO2021155137A1 (en) 2020-01-29 2021-01-29 Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof

Publications (2)

Publication Number Publication Date
JP2023513004A JP2023513004A (ja) 2023-03-30
JP2023513004A5 true JP2023513004A5 (enExample) 2024-02-02

Family

ID=74673421

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022545976A Pending JP2023513004A (ja) 2020-01-29 2021-01-29 眼科遺伝子療法のための改変されたアデノ随伴ウイルスカプシドタンパク質およびその使用方法

Country Status (15)

Country Link
US (1) US20210261625A1 (enExample)
EP (1) EP4097121A1 (enExample)
JP (1) JP2023513004A (enExample)
KR (1) KR20220133941A (enExample)
CN (1) CN115989234A (enExample)
AR (1) AR121228A1 (enExample)
AU (1) AU2021213786A1 (enExample)
BR (1) BR112022014852A2 (enExample)
CA (1) CA3165019A1 (enExample)
CO (1) CO2022010227A2 (enExample)
IL (1) IL294868A (enExample)
MX (1) MX2022009252A (enExample)
PH (1) PH12022551878A1 (enExample)
TW (1) TW202142552A (enExample)
WO (1) WO2021155137A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
EP4551253A1 (en) 2022-07-08 2025-05-14 Ospedale San Raffaele S.r.l. Transgene cassettes
EP4303226A1 (en) 2022-07-08 2024-01-10 Ospedale San Raffaele S.r.l. Transgene cassettes and epigenetic silencers for the treatment of disorders
KR102901502B1 (ko) 2024-06-27 2025-12-18 이엔셀 주식회사 안구 세포 또는 조직을 표적하는 펩타이드 및 이를 포함하는 바이러스 캡시드 단백질 및 바이러스 벡터

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
WO1998048009A2 (en) 1997-04-21 1998-10-29 University Of Florida Materials and methods for ribozyme treatment of retinal diseases
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
US7261544B2 (en) 2003-05-21 2007-08-28 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
US20100081707A1 (en) 2008-02-21 2010-04-01 Ali Robin R Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
KR20180000341A (ko) 2009-06-16 2018-01-02 젠자임 코포레이션 재조합 aav 벡터에 대한 개선된 정제 방법
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
WO2012109570A1 (en) * 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
PL3628334T3 (pl) * 2014-03-21 2023-12-18 Genzyme Corporation Terapia genowa w retinopatii barwnikowej
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
CN107073051B (zh) * 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
CA3033856A1 (en) 2016-08-15 2018-02-22 Genzyme Corporation Methods for detecting aav
US20190262373A1 (en) * 2016-08-16 2019-08-29 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
EP3813845A4 (en) * 2018-05-15 2022-10-26 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties

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