JP2020505936A5 - - Google Patents

Download PDF

Info

Publication number
JP2020505936A5
JP2020505936A5 JP2019542561A JP2019542561A JP2020505936A5 JP 2020505936 A5 JP2020505936 A5 JP 2020505936A5 JP 2019542561 A JP2019542561 A JP 2019542561A JP 2019542561 A JP2019542561 A JP 2019542561A JP 2020505936 A5 JP2020505936 A5 JP 2020505936A5
Authority
JP
Japan
Prior art keywords
amino acid
seq
acid sequence
capsid protein
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.)
Granted
Application number
JP2019542561A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020505936A (ja
JP7291397B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2018/018381 external-priority patent/WO2018152333A1/en
Publication of JP2020505936A publication Critical patent/JP2020505936A/ja
Publication of JP2020505936A5 publication Critical patent/JP2020505936A5/ja
Priority to JP2023007075A priority Critical patent/JP7574508B2/ja
Application granted granted Critical
Publication of JP7291397B2 publication Critical patent/JP7291397B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2019542561A 2017-02-15 2018-02-15 血管系を通過する遺伝子移入の方法および組成物 Active JP7291397B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023007075A JP7574508B2 (ja) 2017-02-15 2023-01-20 血管系を通過する遺伝子移入の方法および組成物

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762459286P 2017-02-15 2017-02-15
US62/459,286 2017-02-15
PCT/US2018/018381 WO2018152333A1 (en) 2017-02-15 2018-02-15 Methods and compositions for gene transfer across the vasculature

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023007075A Division JP7574508B2 (ja) 2017-02-15 2023-01-20 血管系を通過する遺伝子移入の方法および組成物

Publications (3)

Publication Number Publication Date
JP2020505936A JP2020505936A (ja) 2020-02-27
JP2020505936A5 true JP2020505936A5 (https=) 2021-03-04
JP7291397B2 JP7291397B2 (ja) 2023-06-15

Family

ID=63169716

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019542561A Active JP7291397B2 (ja) 2017-02-15 2018-02-15 血管系を通過する遺伝子移入の方法および組成物
JP2023007075A Active JP7574508B2 (ja) 2017-02-15 2023-01-20 血管系を通過する遺伝子移入の方法および組成物

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023007075A Active JP7574508B2 (ja) 2017-02-15 2023-01-20 血管系を通過する遺伝子移入の方法および組成物

Country Status (13)

Country Link
US (2) US11905312B2 (https=)
EP (1) EP3583220A4 (https=)
JP (2) JP7291397B2 (https=)
KR (1) KR20190117571A (https=)
CN (2) CN118978574A (https=)
AU (1) AU2018221738B2 (https=)
BR (1) BR112019016769A2 (https=)
CA (1) CA3052829A1 (https=)
EA (1) EA038695B1 (https=)
IL (2) IL312788A (https=)
MX (1) MX2019009720A (https=)
SG (1) SG11201907298WA (https=)
WO (1) WO2018152333A1 (https=)

Families Citing this family (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
BR112018003665A2 (en) 2015-09-28 2018-09-25 The University Of North Carolina At Chapel Hill methods and compositions for antibody evasion viral vectors
MX2020002148A (es) * 2017-08-25 2020-07-20 Ovid Therapeutics Inc Vectores adenoasociados recombinantes.
US20200263199A1 (en) 2017-09-29 2020-08-20 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
US20210363191A1 (en) 2018-04-03 2021-11-25 Stridebio, Inc. Antibody-evading virus vectors
IL278598B2 (en) 2018-05-11 2025-11-01 Massachusetts Eye & Ear Infirmary Altering tissue tropism of adeno-associated viruses
CN112703198B (zh) 2018-07-11 2025-05-30 布里格姆妇女医院 用于跨血脑屏障递送试剂的方法和组合物
WO2020068990A1 (en) 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
KR20210130158A (ko) * 2019-01-31 2021-10-29 오레곤 헬스 앤드 사이언스 유니버시티 Aav 캡시드의 전사 의존적 유도 진화를 사용하는 방법
BR112021015312A2 (pt) 2019-02-04 2021-11-09 Freeline Therapeutics Ltd Polinucleotídeos, partícula viral e composição
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
TW202102526A (zh) * 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
KR20220012231A (ko) * 2019-04-24 2022-02-03 리젠엑스바이오 인크. 완전-인간 번역 후 변형된 항체 치료제
JP7785352B2 (ja) * 2019-08-14 2025-12-15 ユニバーシティー オブ フロリダ リサーチ ファンデーション, インク. 遺伝子治療のためのaavカプシドバリアント
US12570998B2 (en) 2019-09-09 2026-03-10 Massachusetts Eye & Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (AAV) and the AAV receptor (AAVR) for altered bio-distribution of AAV
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
EP4051324A4 (en) * 2019-10-28 2023-11-29 University Of Florida Research Foundation, Incorporated Gene therapy vectors
PH12022551247A1 (en) * 2019-11-22 2023-11-13 Childrens Hospital Philadelphia Adeno-associated viral vector variants
AU2021206256A1 (en) 2020-01-10 2022-07-28 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
MY209860A (en) 2020-02-13 2025-08-07 Tenaya Therapeutics Inc Gene therapy vectors for treating heart disease
TW202142554A (zh) * 2020-02-25 2021-11-16 澳洲兒童醫學研究所 腺相關病毒蛋白殼多肽及載體
AR122013A1 (es) * 2020-05-05 2022-08-03 Univ Duke Composiciones de virus adenoasociados compatibles entre especies y métodos para su uso
EP4200408A1 (en) 2020-08-19 2023-06-28 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of rett syndrome
JP2023545384A (ja) * 2020-10-07 2023-10-30 リジェネックスバイオ インコーポレイテッド 中枢神経系または筋肉送達のための組換えアデノ随伴ウイルス
WO2022076750A2 (en) * 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
US20240060087A1 (en) * 2021-01-08 2024-02-22 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
WO2022155358A1 (en) * 2021-01-13 2022-07-21 Duke University Compositions for and methods of improving fluid flux in the brain
AU2022307365A1 (en) 2021-07-08 2024-02-01 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
US20250281639A1 (en) * 2021-07-14 2025-09-11 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for treating chronic pain and for retrograde transduction of neurons
CN113563430B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
CN113480615B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用
IL310949A (en) * 2021-08-20 2024-04-01 Biocad Joint Stock Co A method for preparing an adapted adeno-associated virus envelope
AR126839A1 (es) * 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta
US20240425549A1 (en) * 2021-08-25 2024-12-26 Children's Medical Research Institute Modified aav capsid polypeptides and vectors
MX2024002379A (es) * 2021-08-25 2024-04-29 Childrens Medical Res Institute Cápsides y vectores de aav modificados.
WO2023060272A2 (en) * 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
CN114516901B (zh) * 2022-03-11 2023-01-17 上海勉亦生物科技有限公司 一种神经系统高亲和性的aav载体及其应用
WO2023201277A1 (en) * 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
WO2024054864A1 (en) 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Cardioprotective heart disease therapies
KR102855674B1 (ko) * 2022-10-06 2025-09-08 주식회사 글루진테라퓨틱스 폐 기관지 특이적 유전자 전달이 가능한 아데노부속바이러스 벡터
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
AU2024294136A1 (en) * 2023-07-20 2026-02-05 Children's Medical Research Institute Modified cardiotropic aav capsid polypeptides and vectors
KR102868933B1 (ko) * 2023-11-08 2025-10-14 주식회사 글루진테라퓨틱스 성상세포 특이적 유전자 전달이 가능한 아데노부속바이러스 벡터
KR102844372B1 (ko) * 2023-12-06 2025-08-11 주식회사 글루진테라퓨틱스 뇌 성상교세포 특이적 유전자 전달이 가능한 아데노부속바이러스 변이체와 이의 응용
WO2025147741A1 (en) * 2024-01-11 2025-07-17 Children's Medical Research Institute Aav capsids and vectors for transduction of cells
CN119405806A (zh) * 2024-10-25 2025-02-11 南通大学 小肌营养蛋白域含2抑制剂在制备促进神经生长药物中的应用

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2340230T3 (es) 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
DE19933288A1 (de) 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
WO2004027019A2 (en) * 2002-05-01 2004-04-01 University Of Florida Research Foundation, Inc. Improved raav expression systems for genetic modification of specific capsid proteins
EP2396343B1 (en) * 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
JP6348064B2 (ja) * 2011-11-22 2018-06-27 ザ チルドレンズ ホスピタル オブ フィラデルフィア 効率の高いトランスジーン送達のためのウイルスベクター
US9636370B2 (en) * 2012-09-28 2017-05-02 The University Of North Carolina At Chapel Hill AAV vectors targeted to oligodendrocytes
WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
WO2017015102A1 (en) * 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells

Similar Documents

Publication Publication Date Title
JP2020505936A5 (https=)
El Andari et al. Production, processing, and characterization of synthetic AAV gene therapy vectors
JP2021516046A5 (https=)
Bossis et al. Cloning of an avian adeno-associated virus (AAAV) and generation of recombinant AAAV particles
Chen et al. Molecular characterization of adeno-associated viruses infecting children
US10746742B2 (en) Methods of viral neutralizing antibody epitope mapping
JP2022190081A5 (https=)
JP2021514659A5 (https=)
IL317938A (en) Recombinant viral vectors with altered tropism and their uses for the targeted introduction of genetic material into human cells
JP2020522269A5 (https=)
US20130059732A1 (en) Aav capsid proteins for nucleic acid transfer
JP2021533795A5 (https=)
JP2021512649A5 (https=)
JP2018522529A5 (https=)
EP2660325A3 (en) AAV vectors and corresponding nucleotide sequences and methods
JP2007507223A5 (https=)
CA2329060A1 (en) Aav5 vector and uses thereof
JPWO2019168961A5 (https=)
JP2021523702A5 (https=)
Cotmore et al. Parvovirus DNA replication
US20230303983A1 (en) High efficiency purification of divergent aav serotypes using aavx affinity chromatography
JP2020533973A (ja) 改変されたホスホリパーゼドメインを含むアデノ随伴ウイルス(aav)
IL292264B1 (en) Aav transfer cassette
JP2021531044A5 (https=)
JPWO2020041498A5 (https=)