JP2023524577A5 - - Google Patents

Info

Publication number
JP2023524577A5
JP2023524577A5 JP2022567634A JP2022567634A JP2023524577A5 JP 2023524577 A5 JP2023524577 A5 JP 2023524577A5 JP 2022567634 A JP2022567634 A JP 2022567634A JP 2022567634 A JP2022567634 A JP 2022567634A JP 2023524577 A5 JP2023524577 A5 JP 2023524577A5
Authority
JP
Japan
Prior art keywords
aav
nucleic acid
particle
vector
capsid protein
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
JP2022567634A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023524577A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/030547 external-priority patent/WO2021226008A1/en
Publication of JP2023524577A publication Critical patent/JP2023524577A/ja
Publication of JP2023524577A5 publication Critical patent/JP2023524577A5/ja
Pending legal-status Critical Current

Links

JP2022567634A 2020-05-05 2021-05-04 改変アデノ随伴ウイルス5カプシドおよびその使用 Pending JP2023524577A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063020139P 2020-05-05 2020-05-05
US63/020,139 2020-05-05
PCT/US2021/030547 WO2021226008A1 (en) 2020-05-05 2021-05-04 Modified adeno-associated virus 5 capsids and uses thereof

Publications (2)

Publication Number Publication Date
JP2023524577A JP2023524577A (ja) 2023-06-12
JP2023524577A5 true JP2023524577A5 (enExample) 2024-05-15

Family

ID=78412297

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022567634A Pending JP2023524577A (ja) 2020-05-05 2021-05-04 改変アデノ随伴ウイルス5カプシドおよびその使用

Country Status (9)

Country Link
US (1) US12209251B2 (enExample)
EP (1) EP4146673A4 (enExample)
JP (1) JP2023524577A (enExample)
KR (1) KR20230034211A (enExample)
CN (1) CN115916986A (enExample)
AU (1) AU2021267856A1 (enExample)
CA (1) CA3177268A1 (enExample)
MX (1) MX2022013819A (enExample)
WO (1) WO2021226008A1 (enExample)

Families Citing this family (9)

* 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
CN113121651B (zh) * 2021-04-19 2023-11-17 信念医药科技(上海)有限公司 低中和抗体腺相关病毒衣壳蛋白
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
US20250084129A1 (en) * 2022-01-10 2025-03-13 University Of Florida Research Foundation, Incorporated Aav5 capsid variants
US20250388626A1 (en) * 2022-07-06 2025-12-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
CN116693633B (zh) * 2023-02-21 2023-12-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN119570747A (zh) * 2024-12-06 2025-03-07 中国科学院深圳先进技术研究院 一种用于基因与细胞治疗的新型腺相关病毒变体库及其构建和应用

Family Cites Families (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4474893A (en) 1981-07-01 1984-10-02 The University of Texas System Cancer Center Recombinant monoclonal antibodies
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
US4816567A (en) 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
US4676980A (en) 1985-09-23 1987-06-30 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Target specific cross-linked heteroantibodies
AU7966987A (en) 1986-09-08 1988-04-07 Applied Biotechnology, Inc. Empty viral capsid vaccines
US4968603A (en) 1986-12-31 1990-11-06 The Regents Of The University Of California Determination of status in neoplastic disease
EP0442926A1 (en) 1988-11-10 1991-08-28 Imperial Cancer Research Technology Limited Polypeptides
US5916563A (en) 1988-11-14 1999-06-29 United States Of America Parvovirus protein presenting capsids
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
ES2026826A6 (es) 1991-03-26 1992-05-01 Ercros Sa Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados.
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5869248A (en) 1994-03-07 1999-02-09 Yale University Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5599706A (en) 1994-09-23 1997-02-04 Stinchcomb; Dan T. Ribozymes targeted to apo(a) mRNA
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
ATE354957T1 (de) 1995-12-15 2006-03-15 Intronn Inc Durch trans-spaltung erhaltene therapeutische molekule
US6083702A (en) 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
WO1998011244A2 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
AU774706B2 (en) 1998-09-22 2004-07-08 Johns Hopkins University, The Methods for large-scale production of recombinant AAV vectors
ATE362542T1 (de) 1998-11-05 2007-06-15 Univ Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
JP2000325085A (ja) 1999-05-21 2000-11-28 Masafumi Matsuo デュシェンヌ型筋ジストロフィー治療剤
US6653467B1 (en) 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
US7201898B2 (en) 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
US6623729B2 (en) 2001-07-09 2003-09-23 Korea Advanced Institute Of Science And Technology Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer
ITRM20020253A1 (it) 2002-05-08 2003-11-10 Univ Roma Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
WO2005047501A1 (en) 2003-02-24 2005-05-26 Johns Hopkins University Molecular vaccines employing nucleic acid encoding anti-apoptotic proteins
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
ES2926774T3 (es) * 2013-03-15 2022-10-28 Univ North Carolina Chapel Hill Métodos y composiciones para vectores AAV de doble unión de glicano
EP3492597A3 (en) * 2013-05-21 2019-08-28 University of Florida Research Foundation, Inc. Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer
EP4344741A3 (en) * 2014-11-21 2024-08-28 The University of North Carolina at Chapel Hill Aav vectors targeted to the central nervous system
US20180030096A1 (en) * 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
US10081659B2 (en) * 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
WO2018064611A1 (en) 2016-09-30 2018-04-05 Baylor College Of Medicine Antibody based gene therapy with tissue-directed expression
WO2019213668A1 (en) 2018-05-04 2019-11-07 Oregon Health & Science University Human anti-aav2 capsid polyclonal antibody epitopes
AU2019287468B2 (en) * 2018-06-12 2024-10-10 The University Of North Carolina At Chapel Hill Synthetic liver-tropic adeno-associated virus capsids and uses thereof
AU2019357039B2 (en) * 2018-10-12 2025-10-09 Genzyme Corporation Generation of improved human pah for treatment of severe PKU by liver-directed gene replacement therapy

Similar Documents

Publication Publication Date Title
JP2023524577A5 (enExample)
JP7731388B2 (ja) アデノ随伴ウイルスを生産するための哺乳動物細胞
JP2023123765A5 (enExample)
JP2023076606A5 (enExample)
JP2022530633A5 (enExample)
Morris et al. The nucleotide sequence of the infectious cloned DNA component of tobacco yellow dwarf virus reveals features of geminiviruses infecting monocotyledonous plants
JP2023134488A5 (enExample)
JP2022530544A5 (enExample)
JP2022530656A5 (enExample)
JP2017536116A5 (enExample)
JP2014239686A5 (enExample)
CN116390934A (zh) 功能性aav衣壳的高通量工程化
JP2022507534A5 (enExample)
JP2008545406A5 (enExample)
CN101511373A (zh) 用于基因治疗的修饰的因子viii和因子ix基因和载体
CN113748122B (zh) 具有脑靶向特性的aav突变体
CA2349838A1 (en) Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
JP2024180560A5 (enExample)
Zhang et al. Two virus-encoded RNA silencing suppressors, P14 of Beet necrotic yellow vein virus and S6 of Rice black streak dwarf virus
JP2022513797A5 (enExample)
CN113039275B (zh) 用于在植物中表达猪圆环病毒样颗粒的重组载体以及使用其的疫苗组合物
JP2023528801A5 (enExample)
JPWO2020218419A5 (enExample)
JP2024517104A5 (enExample)
JP2004501646A5 (enExample)