JP2022526021A5 - - Google Patents

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Publication number
JP2022526021A5
JP2022526021A5 JP2021559877A JP2021559877A JP2022526021A5 JP 2022526021 A5 JP2022526021 A5 JP 2022526021A5 JP 2021559877 A JP2021559877 A JP 2021559877A JP 2021559877 A JP2021559877 A JP 2021559877A JP 2022526021 A5 JP2022526021 A5 JP 2022526021A5
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JP
Japan
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JP2021559877A
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Japanese (ja)
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JPWO2020210713A5 (https=
JP2022526021A (ja
JP7664848B2 (ja
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JP2021559877A 2019-04-10 2020-04-10 リソソーム障害のための遺伝子療法 Active JP7664848B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962831840P 2019-04-10 2019-04-10
US62/831,840 2019-04-10
US202062990246P 2020-03-16 2020-03-16
US62/990,246 2020-03-16
PCT/US2020/027788 WO2020210713A1 (en) 2019-04-10 2020-04-10 Gene therapies for lysosomal disorders

Publications (4)

Publication Number Publication Date
JP2022526021A JP2022526021A (ja) 2022-05-20
JPWO2020210713A5 JPWO2020210713A5 (https=) 2024-01-19
JP2022526021A5 true JP2022526021A5 (https=) 2024-01-19
JP7664848B2 JP7664848B2 (ja) 2025-04-18

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JP2021559877A Active JP7664848B2 (ja) 2019-04-10 2020-04-10 リソソーム障害のための遺伝子療法

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US (2) US11903985B2 (https=)
EP (1) EP3953378A1 (https=)
JP (1) JP7664848B2 (https=)
KR (1) KR20220006527A (https=)
CN (1) CN114026115A (https=)
AU (1) AU2020270984B2 (https=)
BR (1) BR112021019880A2 (https=)
CA (1) CA3136117A1 (https=)
IL (1) IL286901A (https=)
MX (1) MX2021012184A (https=)
WO (1) WO2020210713A1 (https=)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
EP3692075A4 (en) 2017-10-03 2022-02-09 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISEASES
BR112020006671A2 (pt) 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
BR112021015312A2 (pt) 2019-02-04 2021-11-09 Freeline Therapeutics Ltd Polinucleotídeos, partícula viral e composição
PL3953377T3 (pl) 2019-04-10 2026-02-16 Prevail Therapeutics, Inc. Terapie genowe zaburzeń lizosomalnych
IL300407A (en) 2020-08-10 2023-04-01 Prevail Therapeutics Inc Gene therapies for lysosomal disorders
US20240044869A1 (en) * 2020-10-15 2024-02-08 Prevail Therapeutics, Inc. Assay for measuring potency of gene therapy drug product
US20250082686A1 (en) * 2021-07-21 2025-03-13 Aspen Neuroscience, Inc. Aav-based modulation of gba1 and related compositions and uses thereof
EP4377471A4 (en) * 2021-07-28 2025-10-15 Atsena Therapeutics Inc AAV TRANSFER PLASMIDS
WO2023018674A1 (en) * 2021-08-09 2023-02-16 Amicus Therapeutics, Inc. Determination of gene transduction potency in neuron-like cells
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
MX2024009003A (es) * 2022-01-21 2024-09-17 Astrazeneca Ireland Ltd Terapia genica para la enfermedad de gaucher.
WO2023202637A1 (en) 2022-04-19 2023-10-26 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant aav vectors for treating neurodegenerative disorders
CN117018231B (zh) * 2023-08-16 2024-05-10 科辉智药(深圳)新药研究中心有限公司 用于治疗神经病变的基因疗法及其应用
CN121752725A (zh) * 2023-08-23 2026-03-27 上海金珂博生物技术有限公司 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途
WO2025166048A1 (en) * 2024-01-31 2025-08-07 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Synthetic gba1 genes
WO2025229340A1 (en) 2024-05-01 2025-11-06 Spur Therapeutics Limited Methods and compositions for treatment of a synucleinopathy and/or a disease associated with a gcase deficiency

Family Cites Families (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5879680A (en) 1987-12-23 1999-03-09 The United States Of America As Represented By The Department Of Health And Human Services Cloned DNA for synthesizing unique glucocerebrosidase
US6521225B1 (en) 1996-09-06 2003-02-18 Chiron Corporation AAV vectors
ES2340230T3 (es) 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
US6696272B1 (en) 1999-06-02 2004-02-24 Hsc Research & Development Limited Partnership Products and methods for gaucher disease therapy
GB0009887D0 (en) 2000-04-20 2000-06-07 Btg Int Ltd Cytotoxic agents
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
AU2001290984A1 (en) 2000-09-18 2002-04-02 Genzyme Corporation Expression vectors containing hybrid ubiquitin promoters
US7232670B2 (en) 2001-09-28 2007-06-19 St. Jude Children's Research Hospital Targeting proteins to cells expressing mannose receptors via expression in insect cells
US20030133924A1 (en) 2001-12-21 2003-07-17 Novazyme Pharmaceuticals, Inc. Highly phosphorylated acid beta-glucocerebrosidase and methods of treating gaucher's disease
AU2003221733A1 (en) 2002-04-17 2003-11-03 University Of Florida Research Foundation, Inc. Improved raav vectors
ES2562490T3 (es) 2003-05-01 2016-03-04 Genzyme Corporation Terapia génica para trastornos neurometabólicos
WO2006036465A2 (en) 2004-09-03 2006-04-06 University Of Florida Compositions and methods for treating cystic fibrosis
WO2006039253A2 (en) 2004-09-29 2006-04-13 Children's Memorial Hospital Sirna-mediated gene silencing of alpha synuclein
CN101541172A (zh) * 2005-06-08 2009-09-23 阿米库斯治疗学公司 溶酶体酶编码基因突变相关的cns紊乱的治疗
ES2385679T3 (es) 2006-08-24 2012-07-30 Virovek, Inc. Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos
US8962273B2 (en) 2007-05-11 2015-02-24 Genzyme Corporation Methods of producing a secreted protein
EP2154969B1 (en) 2007-05-16 2015-11-18 The Brigham and Women's Hospital, Inc. Treatment of synucleinopathies
WO2009079399A2 (en) 2007-12-14 2009-06-25 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
WO2010129791A1 (en) 2009-05-06 2010-11-11 University Of Medicine And Dentistry Of New Jersey Rna targeting in alpha-synucleinopathies
EP2826860B1 (en) 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
WO2012027558A2 (en) * 2010-08-25 2012-03-01 The Trustees Of Columbia University In The City Of New York OPTIMIZED miRNA CONSTRUCTS
WO2012027713A2 (en) 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
EP2634253B1 (en) 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
US20150352185A1 (en) 2010-11-16 2015-12-10 Denis G. Kay Method for increasing neprilysin expression and activity
CA2835707C (en) 2011-05-09 2019-02-26 Eip Pharma, Llc Compositions and methods for treating alzheimer's disease
JP2016503405A (ja) * 2012-11-05 2016-02-04 ジェンザイム・コーポレーション タンパク質症を処置するための組成物および方法
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
US9347107B2 (en) 2013-05-23 2016-05-24 Norman Z Lai Vector containing multiple nucleotide sequences for the expression of enzymes
CN120174012A (zh) 2013-10-24 2025-06-20 优尼科Ip有限公司 用于基因治疗神经疾病的aav-5假型载体
IL248102B (en) * 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
WO2016081927A2 (en) 2014-11-21 2016-05-26 University Of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
US20170035860A1 (en) 2015-04-02 2017-02-09 Alexander C. Flynn Compositions and methods for treatment of neurogenerative diseases
JP2015180203A (ja) * 2015-04-17 2015-10-15 ザ スクリプス リサーチ インスティテュート タンパク質生産の増強のためのmRNAの一次構造の再操作
EP3292206B8 (en) 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
EP3091087A1 (en) 2015-05-08 2016-11-09 Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol Method for in vitro diagnosis of synucleinopathies using alpha-synuclein gene transcripts
HK1256341A1 (zh) 2015-05-15 2019-09-20 明尼苏达大学董事会 用於治疗性递送到中枢神经系统的腺相关物
WO2017075338A2 (en) 2015-10-29 2017-05-04 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
RU2021102893A (ru) 2015-11-05 2021-03-03 Бамбу Терапьютикс, Инк. Модифицированные гены атаксии фридрейха и векторы для генной терапии
WO2017136536A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
PL3411484T3 (pl) 2016-02-05 2024-02-19 Emory University Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego
WO2017151884A1 (en) 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
KR20230079511A (ko) 2016-04-21 2023-06-07 바이로베크 인코포레이티드 곤충 세포에서의 aav 생산, 및 이를 위한 방법 및 조성물
BR112020006671A2 (pt) 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
EP3692075A4 (en) 2017-10-03 2022-02-09 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISEASES
AU2018354195A1 (en) 2017-10-23 2020-06-04 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
CA3134841A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
US20220211871A1 (en) 2019-04-10 2022-07-07 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders

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