JP7254815B2 - ライソゾーム病の遺伝子治療 - Google Patents

ライソゾーム病の遺伝子治療 Download PDF

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JP7254815B2
JP7254815B2 JP2020540682A JP2020540682A JP7254815B2 JP 7254815 B2 JP7254815 B2 JP 7254815B2 JP 2020540682 A JP2020540682 A JP 2020540682A JP 2020540682 A JP2020540682 A JP 2020540682A JP 7254815 B2 JP7254815 B2 JP 7254815B2
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raav
vector
itr
seq
sequence
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JP2020537543A5 (https=
JP2020537543A (ja
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アベリオビック，アーサ
ヘックマン，ローラ
ライン，エルベ
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Prevail Therapeutics Inc
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JP2020540682A 2017-10-03 2018-10-03 ライソゾーム病の遺伝子治療 Active JP7254815B2 (ja)

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JP2023052515A JP2023086740A (ja) 2017-10-03 2023-03-29 ライソゾーム病の遺伝子治療
JP2024218686A JP2025063034A (ja) 2017-10-03 2024-12-13 ライソゾーム病の遺伝子治療

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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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JP2020537543A JP2020537543A (ja) 2020-12-24
JP2020537543A5 JP2020537543A5 (https=) 2021-11-11
JP7254815B2 true JP7254815B2 (ja) 2023-04-10

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JP2020540682A Active JP7254815B2 (ja) 2017-10-03 2018-10-03 ライソゾーム病の遺伝子治療
JP2023052515A Ceased JP2023086740A (ja) 2017-10-03 2023-03-29 ライソゾーム病の遺伝子治療
JP2024218686A Withdrawn JP2025063034A (ja) 2017-10-03 2024-12-13 ライソゾーム病の遺伝子治療

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JP2024218686A Withdrawn JP2025063034A (ja) 2017-10-03 2024-12-13 ライソゾーム病の遺伝子治療

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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
AU2018346104B2 (en) 2017-10-03 2023-07-27 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
BR112020006661A2 (pt) 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
CN112501208A (zh) 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
KR20210148101A (ko) 2019-02-04 2021-12-07 프리라인 테라퓨틱스 리미티드 폴리뉴클레오티드
JP7664848B2 (ja) 2019-04-10 2025-04-18 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害のための遺伝子療法
CA3136004A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
US20220298528A1 (en) * 2019-08-12 2022-09-22 UCB Biopharma SRL Viral particles for use in treating synucleinopathies such as parkinson's diseases by gene therapy
MX2022004812A (es) * 2019-10-22 2023-02-23 Applied Genetic Tech Corporation Sistemas de virus adeno-asociados (aav) para el tratamiento de enfermedades o trastornos neurodegenerativos asociados a la progranulina.
JP2021083987A (ja) * 2019-11-29 2021-06-03 株式会社三洋物産 遊技機
CN116324408A (zh) * 2020-06-18 2023-06-23 J·伦德奎斯特 分泌α-突触核蛋白靶向抗体、颗粒蛋白前体和鞘脂激活蛋白原及两者的复合物以及GDNF的细胞系
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
KR20230042513A (ko) 2020-07-29 2023-03-28 프리라인 테라퓨틱스 리미티드 폴리펩티드
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
AU2021325891A1 (en) 2020-08-10 2023-04-06 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disorders
MX2023004420A (es) * 2020-10-15 2023-07-05 Prevail Therapeutics Inc Composiciones de virus adenoasociados recombinantes y métodos para su producción.
EP4229213A1 (en) * 2020-10-15 2023-08-23 Prevail Therapeutics, Inc. Assay for measuring potency of gene therapy drug product
US20250049955A1 (en) 2021-11-17 2025-02-13 Voyager Therapeutics, Inc. Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2023139496A1 (en) * 2022-01-21 2023-07-27 Pfizer Inc. Gene therapy for gaucher disease
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
WO2024229161A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
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

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030133924A1 (en) 2001-12-21 2003-07-17 Novazyme Pharmaceuticals, Inc. Highly phosphorylated acid beta-glucocerebrosidase and methods of treating gaucher's disease
JP2004514407A (ja) 2000-04-28 2004-05-20 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 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
JP2004516016A (ja) 2000-09-18 2004-06-03 ジェンザイム・コーポレイション ハイブリッドユビキチンプロモーターを含む発現ベクター
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
WO2016081927A2 (en) 2014-11-21 2016-05-26 University Of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors

Family Cites Families (43)

* 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
WO1998009524A1 (en) 1996-09-06 1998-03-12 Chiron Corporation Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
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
AU2003221733A1 (en) 2002-04-17 2003-11-03 University Of Florida Research Foundation, Inc. Improved raav vectors
EP1620133B1 (en) 2003-05-01 2015-12-09 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
CA2580189C (en) 2004-09-29 2013-05-21 Children's Memorial Hospital Sirna-mediated gene silencing of alpha synuclein
HUE031156T2 (en) 2006-06-07 2017-06-28 Genzyme Corp Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
WO2008143797A1 (en) 2007-05-11 2008-11-27 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
US20090176729A1 (en) * 2007-12-14 2009-07-09 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
KR20110126717A (ko) 2009-02-24 2011-11-23 더 스크립스 리서치 인스티튜트 단백질 생성 증강을 위한 ｍｒｎａ １차 구조의 재구조화
US9255266B2 (en) 2009-05-06 2016-02-09 Rutgers, The State University Of New Jersey RNA targeting in alpha-synucleinopathies
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors 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
JP6312436B2 (ja) 2010-11-16 2018-04-18 ニューロダイン ライフ サイエンシズ インコーポレイテッドＮｅｕｒｏｄｙｎ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｉｎｃ． ネプリライシンの発現および活性を増大させるための方法および医薬組成物
EP2814838A1 (en) * 2012-02-19 2014-12-24 Protalix Ltd. Oral unit dosage forms and uses of same for the treatment of gaucher disease
EP3196301B1 (en) 2012-07-11 2018-10-17 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of monogenic diseases
CN115120746A (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
US9574184B2 (en) 2013-09-25 2017-02-21 Children's Hospital Medical Center Lysosomal protein targeting sequence and therapeutic applications of same
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
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
CA2985235A1 (en) 2015-05-07 2016-11-10 Shire Human Genetic Therapies, Inc. 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
MX2018004755A (es) 2015-10-29 2018-12-19 Voyager Therapeutics Inc Entrega de polinucleotidos dirigidos al sistema nervioso central.
SG11201802408RA (en) * 2015-11-05 2018-05-30 Bamboo Therapeutics Inc Modified friedreich ataxia genes and vectors for gene therapy
CA3011939A1 (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
EP3411484B1 (en) 2016-02-05 2023-10-04 Emory University Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid
US20190328906A1 (en) 2016-03-02 2019-10-31 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
CN112501208A (zh) 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
BR112020006661A2 (pt) 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
AU2018346104B2 (en) 2017-10-03 2023-07-27 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
BR112020008033A2 (pt) 2017-10-23 2020-10-27 Prevail Therapeutics, Inc. terapias gênicas para doença neurodegenerativa
US20220211871A1 (en) 2019-04-10 2022-07-07 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
JP7664848B2 (ja) 2019-04-10 2025-04-18 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害のための遺伝子療法
WO2020210615A1 (en) 2019-04-10 2020-10-15 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

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004514407A (ja) 2000-04-28 2004-05-20 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 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
JP2004516016A (ja) 2000-09-18 2004-06-03 ジェンザイム・コーポレイション ハイブリッドユビキチンプロモーターを含む発現ベクター
US20030133924A1 (en) 2001-12-21 2003-07-17 Novazyme Pharmaceuticals, Inc. Highly phosphorylated acid beta-glucocerebrosidase and methods of treating gaucher'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
WO2016081927A2 (en) 2014-11-21 2016-05-26 University Of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Journal of Virology，1997年，Vol.71, No.4，pp.3077-3082
Protein Expression and Purification，2002年，vol. 26，p. 96-105

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