MX2021012184A - Terapias genicas para trastornos lisosomales. - Google Patents

Terapias genicas para trastornos lisosomales.

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Publication number
MX2021012184A
MX2021012184A MX2021012184A MX2021012184A MX2021012184A MX 2021012184 A MX2021012184 A MX 2021012184A MX 2021012184 A MX2021012184 A MX 2021012184A MX 2021012184 A MX2021012184 A MX 2021012184A MX 2021012184 A MX2021012184 A MX 2021012184A
Authority
MX
Mexico
Prior art keywords
disease
parkinson
gene therapies
expression constructs
lysosomal disorders
Prior art date
Application number
MX2021012184A
Other languages
English (en)
Inventor
Asa Abeliovich
Laura Heckman
Herve Rhinn
Franz Hefti
Original Assignee
Prevail Therapeutics Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Prevail Therapeutics Inc filed Critical Prevail Therapeutics Inc
Publication of MX2021012184A publication Critical patent/MX2021012184A/es

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    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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Abstract

La descripción se refiere a composiciones y métodos para el tratamiento de enfermedades asociadas con una función lisosomal anómala, tales como la enfermedad de Parkinson y la enfermedad de Gaucher. La descripción proporciona construcciones de expresión que comprenden un transgén que codifica beta-glucocerebrosidasa, un ARN inhibidor que se dirige a alfa-sinucleína, o una combinación de los anteriores. La descripción proporciona, además, métodos para tratar la enfermedad de Gaucher, la enfermedad de Parkinson u otras sinucleinopatías mediante la administración de dichas construcciones de expresión a un sujeto que lo necesita.
MX2021012184A 2019-04-10 2020-04-10 Terapias genicas para trastornos lisosomales. MX2021012184A (es)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
MX2021012184A true MX2021012184A (es) 2022-01-24

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MX2021012184A MX2021012184A (es) 2019-04-10 2020-04-10 Terapias genicas para trastornos lisosomales.

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US (2) US11903985B2 (es)
EP (1) EP3953378A1 (es)
JP (1) JP2022526021A (es)
KR (1) KR20220006527A (es)
CN (1) CN114026115A (es)
AU (1) AU2020270984A1 (es)
BR (1) BR112021019880A2 (es)
CA (1) CA3136117A1 (es)
IL (1) IL286901A (es)
MX (1) MX2021012184A (es)
WO (1) WO2020210713A1 (es)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111542549A (zh) 2017-10-03 2020-08-14 普利维尔治疗公司 用于溶酶体障碍的基因疗法
CA3078501A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
AU2018346105C1 (en) 2017-10-03 2023-10-12 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
AU2020273182A1 (en) 2019-04-10 2021-12-02 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
JP2023537903A (ja) * 2020-08-10 2023-09-06 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害に対する遺伝子療法
WO2023004370A1 (en) * 2021-07-21 2023-01-26 Aspen Neuroscience, Inc. Aav-based modulation of gba1 and related compositions and uses thereof
WO2023018674A1 (en) * 2021-08-09 2023-02-16 Amicus Therapeutics, Inc. Determination of gene transduction potency in neuron-like cells
WO2023139496A1 (en) * 2022-01-21 2023-07-27 Pfizer Inc. Gene therapy for gaucher disease
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

Family Cites Families (47)

* 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
EP0933997A4 (en) 1996-09-06 2000-08-16 Chiron Corp METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS
US6491907B1 (en) 1998-11-10 2002-12-10 The University Of North Carolina At Chapel Hill Recombinant parvovirus vectors and method of making
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
AU2001255575B2 (en) 2000-04-28 2006-08-31 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
ATE310096T1 (de) 2000-09-18 2005-12-15 Genzyme Corp Expressionsvektoren mit hybriden ubiquitin- promotoren
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
WO2004098648A1 (en) 2003-05-01 2004-11-18 Genzyme Corporation Gene therapy for neurometabolic disorders
WO2006036465A2 (en) 2004-09-03 2006-04-06 University Of Florida Compositions and methods for treating cystic fibrosis
DE602005015994D1 (de) 2004-09-29 2009-09-24 Childrens Memorial Hospital siRNA-VERMITTELTES GEN-SILENCING VON ALPHA-SYNUKLEIN
CA2611011C (en) * 2005-06-08 2014-01-28 Amicus Therapeutics, Inc. Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes
EP2061891B1 (en) 2006-08-24 2012-04-11 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
EP2160403B1 (en) 2007-05-11 2018-08-08 Genzyme Corporation Methods of producing a secreted protein
CN101754682B (zh) 2007-05-16 2014-11-12 布里格姆妇女医院 突触核蛋白病的治疗
US20090176729A1 (en) 2007-12-14 2009-07-09 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
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
US9290759B2 (en) 2010-08-25 2016-03-22 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
WO2012057363A1 (ja) 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
EP2640407A4 (en) 2010-11-16 2014-07-09 Denis G Kay PROCESS FOR INCREASING THE EXPRESSION AND ACTIVITY OF NEPRILYSIN
AU2012253615B2 (en) 2011-05-09 2017-03-02 Eip Pharma, Llc Compositions and methods for treating Alzheimer's disease
CN104902923A (zh) 2012-11-05 2015-09-09 建新公司 用于治疗蛋白质病的组合物和方法
CN115120745A (zh) 2013-05-15 2022-09-30 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
CN105745326A (zh) 2013-10-24 2016-07-06 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
DK3137497T3 (da) * 2014-05-02 2021-07-12 Genzyme Corp Aav-vektorer til retinal- og cns-genterapi
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
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
US20180289839A1 (en) 2015-05-15 2018-10-11 Regents Of The University Of Minnesota Intranasal therapeutic delivery of adeno-associated virus to central nervous system
MX2018004755A (es) 2015-10-29 2018-12-19 Voyager Therapeutics Inc Entrega de polinucleotidos dirigidos al sistema nervioso central.
PE20181156A1 (es) 2015-11-05 2018-07-19 Bamboo Therapeutics Inc Genes de ataxia de friedreich modificados y vectores para terapia genica
EP3411059A4 (en) 2016-02-02 2019-10-16 University Of Massachusetts METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM
PT3411484T (pt) 2016-02-05 2023-11-17 Univ Emory Injeção de vírus 9 adeno-associado de cadeia simples ou auto-complementar no líquido cefalorraquidiano
EP3423109A4 (en) 2016-03-02 2019-08-14 The Children's Hospital of Philadelphia THERAPY FOR FRONTOTEMPORAL DEMENTIA
IL261665B2 (en) 2016-04-21 2023-11-01 Virovek Inc AVV production in insect cells, methods and preparations thereof
CA3078501A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
AU2018346105C1 (en) 2017-10-03 2023-10-12 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CN111542549A (zh) 2017-10-03 2020-08-14 普利维尔治疗公司 用于溶酶体障碍的基因疗法
AU2018354195A1 (en) 2017-10-23 2020-06-04 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
US20220211871A1 (en) 2019-04-10 2022-07-07 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CA3134841A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders

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