AU2018346105C1 - Gene therapies for lysosomal disorders - Google Patents

Gene therapies for lysosomal disorders Download PDF

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Publication number
AU2018346105C1
AU2018346105C1 AU2018346105A AU2018346105A AU2018346105C1 AU 2018346105 C1 AU2018346105 C1 AU 2018346105C1 AU 2018346105 A AU2018346105 A AU 2018346105A AU 2018346105 A AU2018346105 A AU 2018346105A AU 2018346105 C1 AU2018346105 C1 AU 2018346105C1
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seq
itr
raav
nucleic acid
sequence
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AU2018346105B2 (en
AU2018346105A1 (en
AU2018346105A2 (en
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Asa Abeliovich
Laura Heckman
Herve Rhinn
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Prevail Therapeutics Inc
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Prevail Therapeutics Inc
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Priority to AU2020260491A priority Critical patent/AU2020260491B2/en
Priority to AU2020260485A priority patent/AU2020260485B2/en
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AU2018346105A 2017-10-03 2018-10-03 Gene therapies for lysosomal disorders Active AU2018346105C1 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2020260491A AU2020260491B2 (en) 2017-10-03 2020-10-29 Gene therapies for lysosomal disorders
AU2020260485A AU2020260485B2 (en) 2017-10-03 2020-10-29 Gene therapies for lysosomal disorders

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201762567296P 2017-10-03 2017-10-03
US201762567301P 2017-10-03 2017-10-03
US201762567319P 2017-10-03 2017-10-03
US201762567310P 2017-10-03 2017-10-03
US201762567311P 2017-10-03 2017-10-03
US62/567,319 2017-10-03
US62/567,310 2017-10-03
US62/567,311 2017-10-03
US62/567,301 2017-10-03
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Families Citing this family (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JOP20190248A1 (ar) 2017-04-21 2019-10-20 Amgen Inc بروتينات ربط مولد ضد trem2 واستخداماته
TWI811229B (zh) 2017-08-03 2023-08-11 美商阿列克特有限責任公司 抗trem2抗體及其使用方法
JP7254815B2 (ja) 2017-10-03 2023-04-10 プリベイル セラピューティクス，インコーポレーテッド ライソゾーム病の遺伝子治療
KR102697811B1 (ko) 2017-10-03 2024-08-22 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
CA3078501A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
HRP20240184T1 (hr) 2019-02-04 2024-04-26 Freeline Therapeutics Limited Polinukleotidi
JP7637058B2 (ja) 2019-02-22 2025-02-27 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 Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス
CA3130055A1 (en) * 2019-02-26 2020-09-03 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
PT3953377T (pt) * 2019-04-10 2025-11-27 Prevail Therapeutics Inc Terapias génicas para distúrbios lisossomais
AU2020270984B2 (en) 2019-04-10 2025-11-20 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector
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.
IL293599A (en) * 2019-12-05 2022-08-01 Univ Columbia Retromer stabilization for the treatment of Alzheimer's disease and other neurodegenerative disorders
WO2021163681A2 (en) * 2020-02-14 2021-08-19 The Trustees Of Columbia University In The City Of New York Combination of retromer pharmacological chaperones and exogenous retromer for the treatment of alzheimer's disease and other neurodegenerative diseases and disorders
US20230210941A1 (en) * 2020-05-12 2023-07-06 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
US20230285595A1 (en) * 2020-07-23 2023-09-14 The University Of North Carolina At Chapel Hill Optimized slc13a5 genes and expression cassettes and their use
US20230227802A1 (en) 2020-07-27 2023-07-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
EP4643868A2 (en) 2020-07-29 2025-11-05 Spur Therapeutics Limited Mutated beta-glucocerebrosidase with improved stability
KR20230043181A (ko) * 2020-08-03 2023-03-30 프리베일 테라퓨틱스, 인크. 파킨을 코딩하는 aav 벡터 및 그의 용도
WO2022035903A2 (en) 2020-08-10 2022-02-17 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
IL300409A (en) * 2020-08-10 2023-04-01 Prevail Therapeutics Inc Gene therapies for neurodegenerative disorders
CN116420078A (zh) 2020-10-14 2023-07-11 戴纳立制药公司 用于治疗和监测额颞痴呆的方法
CN116710567A (zh) 2020-10-15 2023-09-05 普利维尔治疗公司 重组腺相关病毒组合物及其产生方法
MX2023004419A (es) 2020-10-15 2023-07-05 Prevail Therapeutics Inc Ensayo para medir la potencia de un producto farmacológico para genoterapia.
JP2024506860A (ja) * 2021-02-01 2024-02-15 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 ニーマンピック病ａ型を治療するための組成物及び方法
WO2022271944A2 (en) * 2021-06-23 2022-12-29 Avrobio, Inc. Compositions and methods for treatment of gaucher disease
WO2023091949A2 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
JP1727698S (ja) * 2021-12-20 2022-10-19 ゴルフボールケース
CN120866357A (zh) * 2022-04-19 2025-10-31 上海天泽云泰生物医药有限公司 用于治疗神经退行性障碍的重组aav载体
US20240226332A9 (en) 2022-10-04 2024-07-11 Eli Lilly And Company Gene therapy for trem2-associated diseases and disorders
EP4599052A1 (en) * 2022-10-08 2025-08-13 Lingyi Biotech Co., Ltd. Polynucleotides for the treatment of disease associated with gcase deficiency
GB202216168D0 (en) 2022-10-31 2022-12-14 UCB Biopharma SRL Route of administration
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
WO2024189094A1 (en) 2023-03-14 2024-09-19 UCB Biopharma SRL Gene therapy
WO2025039622A1 (zh) * 2023-08-23 2025-02-27 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
CN118086341B (zh) * 2024-04-25 2024-08-02 上海凌医生物科技有限公司 在肝脏中高表达人源葡萄糖脑苷脂酶基因的表达框

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008019187A2 (en) * 2006-05-30 2008-02-14 Mayo Foundation For Medical Education And Research Detecting and treating dementia
WO2017151884A1 (en) * 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia

Family Cites Families (84)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS6182035A (ja) * 1984-09-27 1986-04-25 Toshiba Corp 制振装置
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
US5490559A (en) * 1994-07-20 1996-02-13 Dinulescu; Horia A. Heat exchanger with finned partition walls
US20020068285A1 (en) * 1996-01-11 2002-06-06 Frudakis Tony N. Compositions and methods for the therapy and diagnosis of breast cancer
EP0933997A4 (en) 1996-09-06 2000-08-16 Chiron Corp METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS
HUP0103994A3 (en) 1998-09-09 2004-03-01 Myelos Corp San Diego Method of stimulating prosaposin receptor activity
AU6234099A (en) 1998-10-16 2000-05-08 Introgene B.V. Gene therapy of alzheimer's disease by delivery of an encoded apoliprotein
JP2002538770A (ja) 1998-11-10 2002-11-19 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル ウイルスベクターとその製造及び投与の方法
US6696272B1 (en) 1999-06-02 2004-02-24 Hsc Research & Development Limited Partnership Products and methods for gaucher disease therapy
DE19937800B4 (de) * 1999-08-10 2005-06-16 Gea Energietechnik Gmbh Anlage zur Kondensation von Dampf
AU2001253233B2 (en) 2000-04-06 2006-12-07 Kos Pharmaceuticals, Inc. Compounds and methods for lowering cholesterol levels without inducing hypertriglyceridemia
GB0009887D0 (en) 2000-04-20 2000-06-07 Btg Int Ltd Cytotoxic agents
WO2001083692A2 (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
US6691742B1 (en) * 2003-04-25 2004-02-17 Thomas L. Cooper Method and apparatus for supporting a pipe
EP1620133B1 (en) 2003-05-01 2015-12-09 Genzyme Corporation Gene therapy for neurometabolic disorders
ATE414113T1 (de) 2003-11-21 2008-11-15 Basf Se Polyalkylenoxidgruppen und quartäre stickstoffatome enthaltende copolymere
CN101443046A (zh) * 2004-06-23 2009-05-27 建新公司 治疗多囊病的方法和组合物
WO2006036465A2 (en) 2004-09-03 2006-04-06 University Of Florida Compositions and methods for treating cystic fibrosis
ATE439440T1 (de) 2004-09-29 2009-08-15 Childrens Memorial Hospital Sirna-vermitteltes gen-silencing von alpha- synuklein
CN104771402A (zh) 2005-06-08 2015-07-15 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
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
EP2687223B2 (en) 2006-05-30 2024-11-20 Mayo Foundation For Medical Education And Research Detecting and treating dementia
PL2489733T3 (pl) 2006-06-07 2019-08-30 Genzyme Corporation Terapia genowa w stwardnieniu zanikowym bocznym i innych zaburzeniach rdzenia kręgowego
DE102006029773B3 (de) * 2006-06-27 2007-07-12 Gea Energietechnik Gmbh Verfahren zur Errichtung einer Kondensationsanlage
CN101583621A (zh) * 2006-07-20 2009-11-18 诺华有限公司 治疗、诊断或检测癌症的amigo-2抑制剂
JP5721426B2 (ja) 2007-04-05 2015-05-20 ザ ジェイ．ディヴィッド グラッドストン インスティテューツ 神経の過度な興奮を緩和する薬剤を識別する方法
EP3456733A1 (en) 2007-05-11 2019-03-20 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
AU2008268461B2 (en) * 2007-06-22 2015-04-09 Children's Medical Center Corporation Methods and uses thereof of prosaposin
US20090176729A1 (en) 2007-12-14 2009-07-09 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
EP3009143B1 (en) 2008-01-16 2018-10-03 Neurodyn Life Sciences Inc. Progranulin for use in treating parkinson's disease or alzheimer's disease
US9265843B2 (en) * 2008-03-27 2016-02-23 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of BDNF and compositions therefor
BRPI1010868A2 (pt) 2009-05-02 2018-06-12 Genzyme Corp terapia gênica para distúrbios neurodegenerativos
WO2010129791A1 (en) 2009-05-06 2010-11-11 University Of Medicine And Dentistry Of New Jersey Rna targeting in alpha-synucleinopathies
CN102858985A (zh) * 2009-07-24 2013-01-02 西格马-奥尔德里奇有限责任公司 基因组编辑方法
AU2010339794B2 (en) * 2009-12-17 2016-09-22 Children's Medical Center Corporation Saposin-A derived peptides and uses thereof
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
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
EP3081568B1 (en) 2011-05-09 2019-11-13 Eip Pharma, LLC Compositions and methods for treating alzheimer's disease
EP2834260A4 (en) * 2012-04-02 2016-08-10 Moderna Therapeutics Inc MODIFIED POLYNUCLEOTIDES FOR THE PREPARATION OF MEMBRANE PROTEINS
RU2673484C2 (ru) 2012-05-18 2018-11-27 Юниверсити Оф Айова Рисерч Фаундейшн Способы и композиции для лечения амилоидных отложений
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
MX2015005722A (es) 2012-11-05 2016-01-12 Genzyme Corp Composiciones y metodos para el tratamiento de proteinopatias.
CA2909085C (en) 2013-04-08 2023-08-29 University Of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
CA3209821A1 (en) 2013-05-15 2014-11-20 Regents Of The University Of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
US9347107B2 (en) 2013-05-23 2016-05-24 Norman Z Lai Vector containing multiple nucleotide sequences for the expression of enzymes
CA2917569A1 (en) 2013-07-11 2015-01-15 The Trustees Of Columbia University In The City Of New York Micrornas that silence tau expression
US9574184B2 (en) 2013-09-25 2017-02-21 Children's Hospital Medical Center Lysosomal protein targeting sequence and therapeutic applications of same
CA2955086A1 (en) * 2014-08-08 2016-02-11 Alector Llc Anti-trem2 antibodies and methods of use thereof
MA40460A (fr) * 2014-08-11 2016-02-18 Shire Human Genetic Therapies Ciblage lysosomial et utilisation correspondante
GB2558326B (en) * 2014-09-05 2021-01-20 Population Bio Inc Methods and compositions for inhibiting and treating neurological conditions
EP3221456B1 (en) 2014-11-21 2021-09-22 University of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
ES2714130T3 (es) 2015-02-02 2019-05-27 Procter & Gamble Composición detergente
EP3274456B1 (en) 2015-03-25 2020-06-24 Università Degli Studi Di Trento Rna interference mediated therapy for neurodegenerative diseases
US20170035860A1 (en) 2015-04-02 2017-02-09 Alexander C. Flynn Compositions and methods for treatment of neurogenerative diseases
CA3019315A1 (en) * 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
US10967073B2 (en) 2015-05-07 2021-04-06 The Mclean Hospital Corporation 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
PT3294323T (pt) 2015-05-15 2022-01-06 Univ Minnesota Vírus adeno-associado para administração terapêutica ao sistema nervoso central
WO2017058561A1 (en) * 2015-10-02 2017-04-06 The Procter & Gamble Company Cleaning pad, cleaning implement, and a method of improving shine of a hard surface using the cleaning pad or cleaning implement with a cleaning composition
AU2016343979A1 (en) 2015-10-29 2018-05-10 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
KR20210005994A (ko) 2015-11-05 2021-01-15 뱀부 테라퓨틱스 인코포레이티드 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터
US11826433B2 (en) 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
US12070510B2 (en) 2016-02-05 2024-08-27 Emory University Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid
WO2017147509A1 (en) * 2016-02-25 2017-08-31 Marco Colonna Compositions comprising trem2 and methods of use thereof
EP3228690B1 (en) * 2016-04-08 2020-05-13 The Procter and Gamble Company Automatic dishwashing cleaning composition
TWI722310B (zh) 2017-08-03 2021-03-21 美商航海家醫療公司 用於aav之遞送之組合物及方法
KR102697811B1 (ko) 2017-10-03 2024-08-22 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
JP7254815B2 (ja) 2017-10-03 2023-04-10 プリベイル セラピューティクス，インコーポレーテッド ライソゾーム病の遺伝子治療
CA3078501A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CN111819281B (zh) 2017-10-23 2024-12-31 普利维尔治疗公司 用于神经变性疾病的基因疗法
US20200283800A1 (en) 2017-10-23 2020-09-10 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative diseases
MX2021006253A (es) 2018-11-28 2021-09-21 Prevail Therapeutics Inc Terapias génicas para enfermedad neurodegenerativa.
WO2020210615A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
PT3953377T (pt) 2019-04-10 2025-11-27 Prevail Therapeutics Inc Terapias génicas para distúrbios lisossomais
AU2020270984B2 (en) 2019-04-10 2025-11-20 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
US20220211871A1 (en) 2019-04-10 2022-07-07 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008019187A2 (en) * 2006-05-30 2008-02-14 Mayo Foundation For Medical Education And Research Detecting and treating dementia
WO2017151884A1 (en) * 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia

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