JPWO2022183052A5 - - Google Patents

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Publication number
JPWO2022183052A5
JPWO2022183052A5 JP2023552146A JP2023552146A JPWO2022183052A5 JP WO2022183052 A5 JPWO2022183052 A5 JP WO2022183052A5 JP 2023552146 A JP2023552146 A JP 2023552146A JP 2023552146 A JP2023552146 A JP 2023552146A JP WO2022183052 A5 JPWO2022183052 A5 JP WO2022183052A5
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JP
Japan
Prior art keywords
capsid
raav
vector
promoter
itr
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
JP2023552146A
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English (en)
Japanese (ja)
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JP2024509792A5 (https=
JP2024509792A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/017998 external-priority patent/WO2022183052A1/en
Publication of JP2024509792A publication Critical patent/JP2024509792A/ja
Publication of JP2024509792A5 publication Critical patent/JP2024509792A5/ja
Publication of JPWO2022183052A5 publication Critical patent/JPWO2022183052A5/ja
Withdrawn legal-status Critical Current

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JP2023552146A 2021-02-26 2022-02-25 ファブリー病の治療のための組成物及び方法 Withdrawn JP2024509792A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163154485P 2021-02-26 2021-02-26
US63/154,485 2021-02-26
PCT/US2022/017998 WO2022183052A1 (en) 2021-02-26 2022-02-25 Composition and methods for the treatment of fabry disease

Publications (3)

Publication Number Publication Date
JP2024509792A JP2024509792A (ja) 2024-03-05
JP2024509792A5 JP2024509792A5 (https=) 2025-03-03
JPWO2022183052A5 true JPWO2022183052A5 (https=) 2025-03-03

Family

ID=80786597

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023552146A Withdrawn JP2024509792A (ja) 2021-02-26 2022-02-25 ファブリー病の治療のための組成物及び方法

Country Status (17)

Country Link
US (1) US20240175054A1 (https=)
EP (1) EP4298227A1 (https=)
JP (1) JP2024509792A (https=)
KR (1) KR20230150836A (https=)
CN (1) CN117321212A (https=)
AR (1) AR124981A1 (https=)
AU (1) AU2022227017A1 (https=)
BR (1) BR112023016983A2 (https=)
CA (1) CA3209673A1 (https=)
CL (1) CL2023002530A1 (https=)
CO (1) CO2023011012A2 (https=)
EC (1) ECSP23072174A (https=)
IL (1) IL305440A (https=)
MX (1) MX2023009978A (https=)
PE (1) PE20240806A1 (https=)
TW (1) TW202302855A (https=)
WO (1) WO2022183052A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025529884A (ja) * 2022-08-25 2025-09-09 武田薬品工業株式会社 ファブリー病の治療に使用するための組成物
CN121002183A (zh) * 2023-04-17 2025-11-21 杭州复因生物科技有限公司 用于基因治疗的组合物和方法
WO2024236544A1 (en) 2023-05-17 2024-11-21 Takeda Pharmaceutical Company Limited Pet tracer compounds for tracing alpha-galactosidase a and reaction intermediates, and methods of use

Family Cites Families (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
WO1998046728A1 (en) 1997-04-14 1998-10-22 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant aav product
WO1999061643A1 (en) 1998-05-27 1999-12-02 University Of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient
ES2340230T3 (es) 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
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
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
CN1856576B (zh) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
WO2006110689A2 (en) 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
US9249425B2 (en) 2011-05-16 2016-02-02 The Trustees Of The University Of Pennslyvania Proviral plasmids and production of recombinant adeno-associated virus
EP3794131A4 (en) * 2018-05-15 2022-01-26 University of Massachusetts RAAV VECTORS ENCODING FOR LYSOSOMAL BETA-GALACTOSIDASE (GLB1) AND CATHEPSIN A
AR116624A1 (es) * 2018-10-10 2021-05-26 Amicus Therapeutics Inc Composiciones de polipéptidos estabilizados por enlaces disulfuro y métodos de uso
US12454701B2 (en) * 2018-12-05 2025-10-28 Abeona Therapeutics Inc. Recombinant adeno-associated viral vector for gene delivery
SG11202106348QA (en) * 2018-12-20 2021-07-29 Codexis Inc Human alpha-galactosidase variants
JP7716338B2 (ja) * 2019-01-04 2025-07-31 サンガモ セラピューティクス, インコーポレイテッド ファブリー病の処置のための方法及び組成物
AU2021356684A1 (en) * 2020-10-09 2023-05-11 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease

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