KR102693318B1 - 리소좀 장애를 위한 유전자 요법 - Google Patents

리소좀 장애를 위한 유전자 요법 Download PDF

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KR102693318B1
KR102693318B1 KR1020207012188A KR20207012188A KR102693318B1 KR 102693318 B1 KR102693318 B1 KR 102693318B1 KR 1020207012188 A KR1020207012188 A KR 1020207012188A KR 20207012188 A KR20207012188 A KR 20207012188A KR 102693318 B1 KR102693318 B1 KR 102693318B1
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itr
raav
vector
nucleic acid
sequence
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KR20200078512A (ko
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아사 아벨리오비치
로라 헤크만
허브 린
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프리베일 테라퓨틱스, 인크.
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    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
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KR1020207012188A 2017-10-03 2018-10-03 리소좀 장애를 위한 유전자 요법 Active KR102693318B1 (ko)

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Application Number Priority Date Filing Date Title
US201762567296P 2017-10-03 2017-10-03
US62/567,296 2017-10-03
PCT/US2018/054225 WO2019070893A1 (en) 2017-10-03 2018-10-03 GENE THERAPIES FOR LYSOSOMAL DISORDERS

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KR20200078512A KR20200078512A (ko) 2020-07-01
KR102693318B1 true KR102693318B1 (ko) 2024-08-07

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US (4) US11802294B2 (https=)
EP (1) EP3692075A4 (https=)
JP (3) JP7254815B2 (https=)
KR (1) KR102693318B1 (https=)
CN (1) CN111542549A (https=)
AU (2) AU2018346104B2 (https=)
BR (1) BR112020006633A2 (https=)
CA (1) CA3078371A1 (https=)
IL (2) IL278868B2 (https=)
MX (2) MX2020003557A (https=)
WO (1) WO2019070893A1 (https=)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
BR112021015312A2 (pt) 2019-02-04 2021-11-09 Freeline Therapeutics Ltd Polinucleotídeos, partícula viral e composição
CA3136117A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
PL3953377T3 (pl) 2019-04-10 2026-02-16 Prevail Therapeutics, Inc. Terapie genowe zaburzeń lizosomalnych
WO2021028299A1 (en) * 2019-08-12 2021-02-18 Fundacion Para La Investigacion Medica Aplicada Viral particles for use in treating synucleinopathies such as parkinson's diseases by gene therapy
IL292382A (en) * 2019-10-22 2022-06-01 Applied Genetic Tech Corporation Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegenerative diseases or disorders
JP2021083987A (ja) * 2019-11-29 2021-06-03 株式会社三洋物産 遊技機
EP4168795A4 (en) * 2020-06-18 2024-09-04 Lundkvist, Johan CELL LINES SECRETING ANTIBODIES TARGETING ALPHA-SYNUCLEIN, PROGRANULIN AND PROSAPOSIN AND A COMPLEX OF THE TWO, AND GDNF
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
EP4118200B1 (en) 2020-07-29 2025-10-08 Spur Therapeutics Limited Mutated beta-glucocerebrosidase with improved stability
IL300407A (en) * 2020-08-10 2023-04-01 Prevail Therapeutics Inc Gene therapies for lysosomal disorders
US20230346979A1 (en) 2020-08-10 2023-11-02 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disorders
KR20230088393A (ko) * 2020-10-15 2023-06-19 프리베일 테라퓨틱스, 인크. 재조합 아데노 연관 바이러스 조성물 및 이의 제조 방법
US20240044869A1 (en) 2020-10-15 2024-02-08 Prevail Therapeutics, Inc. Assay for measuring potency of gene therapy drug product
EP4433490A2 (en) 2021-11-17 2024-09-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
MX2024009003A (es) * 2022-01-21 2024-09-17 Astrazeneca Ireland Ltd Terapia genica para la enfermedad de gaucher.
AU2023427408A1 (en) 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
CN121127597A (zh) 2023-05-03 2025-12-12 沃雅戈治疗公司 用于治疗与葡糖苷酰鞘氨醇酶β1缺陷相关的病症的组合物及方法
WO2025038805A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025063407A1 (ko) * 2023-09-22 2025-03-27 주식회사 스탠드업테라퓨티스 신규한 파킨슨 병 치료제

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
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
WO2007146046A2 (en) 2006-06-07 2007-12-21 Genzyme Corporation Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
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
US8853179B2 (en) 2009-02-24 2014-10-07 The Scripps Research Institute Reengineering mRNA primary structure for enhanced protein production
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
US20150352185A1 (en) 2010-11-16 2015-12-10 Denis G. Kay Method for increasing neprilysin expression and activity
WO2013121405A1 (en) 2012-02-19 2013-08-22 Protalix Ltd. Oral unit dosage forms and uses of same for the treatment of gaucher disease
DK3444342T3 (da) 2012-07-11 2020-08-24 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til behandlingen af lysosomale aflejringssygdomme
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
US9574184B2 (en) 2013-09-25 2017-02-21 Children's Hospital Medical Center Lysosomal protein targeting sequence and therapeutic applications of same
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
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
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
US20220211871A1 (en) 2019-04-10 2022-07-07 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CA3136117A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CA3134841A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
IL300407A (en) 2020-08-10 2023-04-01 Prevail Therapeutics Inc Gene therapies for lysosomal disorders

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