JP2023513004A5 - - Google Patents

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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
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JP
Japan
Prior art keywords
amino acid
sequence
capsid protein
raav
nucleic acid
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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.)
Granted
Application number
JP2022545976A
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English (en)
Japanese (ja)
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JP7866941B2 (ja
JP2023513004A (ja
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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
Application granted granted Critical
Publication of JP7866941B2 publication Critical patent/JP7866941B2/ja
Active legal-status Critical Current
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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 Active JP7866941B2 (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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2026082295A Division JP2026133539A (ja) 2020-01-29 2026-05-15 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

Publications (3)

Publication Number Publication Date
JP2023513004A JP2023513004A (ja) 2023-03-30
JP2023513004A5 true JP2023513004A5 (enExample) 2024-02-02
JP7866941B2 JP7866941B2 (ja) 2026-05-28

Family

ID=74673421

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022545976A Active JP7866941B2 (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 (14)

Country Link
US (1) US20210261625A1 (enExample)
EP (1) EP4097121A1 (enExample)
JP (1) JP7866941B2 (enExample)
KR (1) KR20220133941A (enExample)
CN (1) CN115989234A (enExample)
AR (1) AR121228A1 (enExample)
AU (2) 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)
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
US20260000790A1 (en) 2022-07-08 2026-01-01 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
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
NZ500427A (en) 1997-04-21 2002-12-20 Univ Florida The use of ribozyme treatment for retinal diseases
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6995006B2 (en) 1997-09-05 2006-02-07 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
JP4559429B2 (ja) 2003-05-21 2010-10-06 ジェンザイム・コーポレーション 空キャプシドを実質的に含まない組換えaavビリオン調製物を生成するための方法
DK2623605T3 (en) 2007-04-09 2019-03-25 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
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
JP5956331B2 (ja) 2009-06-16 2016-07-27 ジェンザイム・コーポレーション 組換えaavベクターの改良された精製方法
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
CA2826273C (en) * 2011-02-10 2021-11-02 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
PT3628334T (pt) * 2014-03-21 2023-09-26 Genzyme Corp Terapia genética para retinopatia pigmentar
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
WO2016065001A1 (en) * 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
HUE053747T2 (hu) 2016-08-15 2021-07-28 Genzyme Corp Eljárások AAV kimutatására
CA3033125A1 (en) * 2016-08-16 2018-02-22 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
WO2019221992A1 (en) * 2018-05-15 2019-11-21 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties

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