JP2022512589A5 - - Google Patents

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Publication number
JP2022512589A5
JP2022512589A5 JP2021518190A JP2021518190A JP2022512589A5 JP 2022512589 A5 JP2022512589 A5 JP 2022512589A5 JP 2021518190 A JP2021518190 A JP 2021518190A JP 2021518190 A JP2021518190 A JP 2021518190A JP 2022512589 A5 JP2022512589 A5 JP 2022512589A5
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JP
Japan
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JP2021518190A
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Japanese (ja)
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JP2022512589A (ja
JP7534290B2 (ja
JPWO2020072354A5 (https=
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Priority claimed from PCT/US2019/053797 external-priority patent/WO2020072354A1/en
Publication of JP2022512589A publication Critical patent/JP2022512589A/ja
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Publication of JPWO2020072354A5 publication Critical patent/JPWO2020072354A5/ja
Priority to JP2024125570A priority Critical patent/JP2024161415A/ja
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JP2021518190A 2018-10-01 2019-09-30 Gm1ガングリオシドーシスの治療に有用な組成物 Active JP7534290B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024125570A JP2024161415A (ja) 2018-10-01 2024-08-01 Gm1ガングリオシドーシスの治療に有用な組成物

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862739811P 2018-10-01 2018-10-01
US62/739,811 2018-10-01
US201962835178P 2019-04-17 2019-04-17
US62/835,178 2019-04-17
PCT/US2019/053797 WO2020072354A1 (en) 2018-10-01 2019-09-30 Compositions useful for treating gm1 gangliosidosis

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024125570A Division JP2024161415A (ja) 2018-10-01 2024-08-01 Gm1ガングリオシドーシスの治療に有用な組成物

Publications (4)

Publication Number Publication Date
JP2022512589A JP2022512589A (ja) 2022-02-07
JP2022512589A5 true JP2022512589A5 (https=) 2022-10-06
JPWO2020072354A5 JPWO2020072354A5 (https=) 2022-10-06
JP7534290B2 JP7534290B2 (ja) 2024-08-14

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ID=69165478

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JP2021518190A Active JP7534290B2 (ja) 2018-10-01 2019-09-30 Gm1ガングリオシドーシスの治療に有用な組成物
JP2024125570A Pending JP2024161415A (ja) 2018-10-01 2024-08-01 Gm1ガングリオシドーシスの治療に有用な組成物

Family Applications After (1)

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JP2024125570A Pending JP2024161415A (ja) 2018-10-01 2024-08-01 Gm1ガングリオシドーシスの治療に有用な組成物

Country Status (16)

Country Link
US (1) US12594327B2 (https=)
EP (1) EP3860659A1 (https=)
JP (2) JP7534290B2 (https=)
KR (1) KR20210071017A (https=)
CN (1) CN113438954A (https=)
AU (1) AU2019351815A1 (https=)
BR (1) BR112021006060A2 (https=)
CA (1) CA3114175A1 (https=)
CL (1) CL2021000798A1 (https=)
CO (1) CO2021005443A2 (https=)
IL (1) IL281923A (https=)
MX (1) MX2021003869A (https=)
PE (1) PE20211596A1 (https=)
SA (1) SA521421639B1 (https=)
SG (1) SG11202102942VA (https=)
WO (1) WO2020072354A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113438954A (zh) 2018-10-01 2021-09-24 宾夕法尼亚州大学信托人 可用于治疗gm1神经节苷脂病的组合物
MX2022009462A (es) * 2020-02-02 2022-11-10 Univ Pennsylvania Composiciones útiles para tratar la gangliosidosis gm1.
AR122013A1 (es) * 2020-05-05 2022-08-03 Univ Duke Composiciones de virus adenoasociados compatibles entre especies y métodos para su uso
CN116926047A (zh) * 2022-03-31 2023-10-24 北京锦篮基因科技有限公司 功能性β-半乳糖苷酶变体、AAV介导的人β-半乳糖苷酶表达载体及其用途
WO2024040258A2 (en) * 2022-08-19 2024-02-22 Codexis, Inc. Engineered beta- galactosidase polypeptides
CN120035662A (zh) * 2022-10-12 2025-05-23 新型医药公司 用于治疗、预防或改善GM1神经节苷脂沉积症或Morquio综合征B的药物组合物及其施用方法

Family Cites Families (42)

* 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
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
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
EP1660970A4 (en) 2003-08-01 2007-02-14 Dna Twopointo Inc SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY
US8005620B2 (en) 2003-08-01 2011-08-23 Dna Twopointo Inc. Systems and methods for biopolymer engineering
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
PL3252161T3 (pl) * 2007-06-06 2022-03-07 Genzyme Corporation Terapia genowa dla lizosomalnych chorób spichrzeniowych
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US20130023033A1 (en) 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
AU2013243948A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
CN105377039A (zh) 2013-05-15 2016-03-02 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
BR112017005892A2 (pt) 2014-09-24 2017-12-12 Hope City variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma
CA3019315A1 (en) 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
WO2017075338A2 (en) 2015-10-29 2017-05-04 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
US20190060359A1 (en) 2015-11-02 2019-02-28 Neuraltus Pharmaceuticals, Inc. Treatment of neurodegenerative disease with sodium chlorite
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
EP3411488A1 (en) 2016-02-03 2018-12-12 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
PL3411484T3 (pl) 2016-02-05 2024-02-19 Emory University Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego
WO2018045347A1 (en) 2016-09-02 2018-03-08 Spark Therapeutics, Inc. Methods and vectors for treating cns disorders
EP3541946A1 (en) 2016-11-15 2019-09-25 Regents Of The University Of Minnesota Method for improving neurological function in mpsi and mpsii and other neurological disorders
JP7455579B2 (ja) 2017-02-28 2024-03-26 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 新規アデノ随伴ウイルス(aav)クレードfベクター及びその用途
EP3794131A4 (en) 2018-05-15 2022-01-26 University of Massachusetts RAAV VECTORS ENCODING FOR LYSOSOMAL BETA-GALACTOSIDASE (GLB1) AND CATHEPSIN A
CN113438954A (zh) 2018-10-01 2021-09-24 宾夕法尼亚州大学信托人 可用于治疗gm1神经节苷脂病的组合物
US12448629B2 (en) 2018-10-05 2025-10-21 University Of Massachusetts rAAV vectors for the treatment of GM1 and GM2 gangliosidosis
US12454701B2 (en) 2018-12-05 2025-10-28 Abeona Therapeutics Inc. Recombinant adeno-associated viral vector for gene delivery
MX2022009462A (es) 2020-02-02 2022-11-10 Univ Pennsylvania Composiciones útiles para tratar la gangliosidosis gm1.

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