MX2020003557A - Terapias genicas para los trastornos lisosomales. - Google Patents

Terapias genicas para los trastornos lisosomales.

Info

Publication number
MX2020003557A
MX2020003557A MX2020003557A MX2020003557A MX2020003557A MX 2020003557 A MX2020003557 A MX 2020003557A MX 2020003557 A MX2020003557 A MX 2020003557A MX 2020003557 A MX2020003557 A MX 2020003557A MX 2020003557 A MX2020003557 A MX 2020003557A
Authority
MX
Mexico
Prior art keywords
disease
gene therapies
expression constructs
disclosure provides
lysosomal
Prior art date
Application number
MX2020003557A
Other languages
English (en)
Spanish (es)
Inventor
Asa Abeliovich
Laura Heckman
Herve Rhinn
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 MX2020003557A publication Critical patent/MX2020003557A/es

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/861—Adenoviral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/141—MicroRNAs, miRNAs
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
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    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vectore
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Animal Behavior & Ethology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MX2020003557A 2017-10-03 2018-10-03 Terapias genicas para los trastornos lisosomales. MX2020003557A (es)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
MX2020003557A true MX2020003557A (es) 2020-10-07

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Application Number Title Priority Date Filing Date
MX2020003557A MX2020003557A (es) 2017-10-03 2018-10-03 Terapias genicas para los trastornos lisosomales.
MX2020011745A MX2020011745A (es) 2017-10-03 2020-07-13 Terapias genicas para los trastornos lisosomales.

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Application Number Title Priority Date Filing Date
MX2020011745A MX2020011745A (es) 2017-10-03 2020-07-13 Terapias genicas para los trastornos lisosomales.

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US (4) US11802294B2 (enExample)
EP (1) EP3692075A4 (enExample)
JP (3) JP7254815B2 (enExample)
KR (1) KR102693318B1 (enExample)
CN (1) CN111542549A (enExample)
AU (2) AU2018346104B2 (enExample)
BR (1) BR112020006633A2 (enExample)
CA (1) CA3078371A1 (enExample)
IL (2) IL273776B2 (enExample)
MX (2) MX2020003557A (enExample)
WO (1) WO2019070893A1 (enExample)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US11802294B2 (en) 2017-10-03 2023-10-31 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
FI3850089T3 (fi) 2019-02-04 2024-02-14 Freeline Therapeutics Ltd Polynukleotidejä
DK3953377T3 (da) 2019-04-10 2025-12-01 Prevail Therapeutics Inc Genterapier til lysosomale lidelser
WO2020210713A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
JP2023500011A (ja) * 2019-08-12 2023-01-04 ファンダシオン パラ ラ インベスティガシオン メディカ アプリカダ 遺伝子療法によるパーキンソン病などのシヌクレイノパチーの治療に使用するためのウイルス粒子
CN115715327A (zh) * 2019-10-22 2023-02-24 应用遗传科技公司 用于治疗颗粒蛋白前体相关的神经退行性疾病或病症的腺伴随病毒(aav)系统
JP2021083987A (ja) * 2019-11-29 2021-06-03 株式会社三洋物産 遊技機
WO2021258074A2 (en) * 2020-06-18 2021-12-23 Lundkvist Johan Cell lines secreting alpha-synuclein targeting antibodies, progranulin and prosaposin and a complex of both, and gdnf
CA3190309A1 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
CA3189801A1 (en) 2020-07-29 2022-02-03 Freeline Therapeutics Limited Polypeptide
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
KR20230066360A (ko) 2020-08-10 2023-05-15 프리베일 테라퓨틱스, 인크. 신경퇴행성 장애를 위한 유전자 요법
KR20230088393A (ko) * 2020-10-15 2023-06-19 프리베일 테라퓨틱스, 인크. 재조합 아데노 연관 바이러스 조성물 및 이의 제조 방법
JP2023545835A (ja) * 2020-10-15 2023-10-31 プリベイル セラピューティクス，インコーポレーテッド 遺伝子療法製剤の効力を測定するためのアッセイ
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
EP4465998A1 (en) * 2022-01-21 2024-11-27 Astrazeneca Ireland Limited 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
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
KR20250046126A (ko) * 2023-09-22 2025-04-02 주식회사 스탠드업테라퓨티스 신규한 파킨슨 병 치료제

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
EP0933997A4 (en) 1996-09-06 2000-08-16 Chiron Corp METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION 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
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
EP1319082B1 (en) 2000-09-18 2005-11-16 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
US20060292117A1 (en) 2002-04-17 2006-12-28 Loiler Scott A Improved rAAv vectors
PL1620133T3 (pl) 2003-05-01 2016-05-31 Genzyme Corp Terapia genowa dla zaburzeń neurometabolicznych
WO2006036465A2 (en) 2004-09-03 2006-04-06 University Of Florida Compositions and methods for treating cystic fibrosis
EP1799826B1 (en) 2004-09-29 2009-08-12 Children's Memorial Hospital siRNA-MEDIATED GENE SILENCING OF ALPHA SYNUCLEIN
ES2596885T3 (es) 2006-06-07 2017-01-12 Genzyme Corporation Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la medula espinal
WO2008143797A1 (en) 2007-05-11 2008-11-27 Genzyme Corporation Methods of producing a secreted protein
HUE027278T2 (en) 2007-05-16 2016-11-28 Brigham & Womens Hospital Inc Treatment of synucleinopathies
WO2009079399A2 (en) 2007-12-14 2009-06-25 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
US9255266B2 (en) 2009-05-06 2016-02-09 Rutgers, The State University Of New Jersey RNA targeting in alpha-synucleinopathies
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
EP2640407A4 (en) 2010-11-16 2014-07-09 Denis G Kay PROCESS FOR INCREASING THE EXPRESSION AND ACTIVITY OF NEPRILYSIN
WO2013121405A1 (en) * 2012-02-19 2013-08-22 Protalix Ltd. Oral unit dosage forms and uses of same for the treatment of gaucher disease
BR112015009746A2 (pt) * 2012-11-05 2017-08-15 Genzyme Corp Agente que aumenta atividade de glicocerebrosidase em mamíferos
RU2692251C2 (ru) 2013-05-15 2019-06-24 Риджентс Оф Зэ Юниверсити Оф Миннесота Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему
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
US10900053B2 (en) * 2014-11-21 2021-01-26 University Of Florida Research Foundation, Incorporated 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
AU2016256895B2 (en) 2015-05-07 2022-05-26 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
WO2016187017A1 (en) 2015-05-15 2016-11-24 Mcivor R Scott Adeno-associated for therapeutic delivery to central nervous system
WO2017075338A2 (en) 2015-10-29 2017-05-04 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
CN108348621A (zh) * 2015-11-05 2018-07-31 竹治疗股份有限公司 用于基因治疗的经修饰的弗里德赖希共济失调基因及载体
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
IL300254B2 (en) 2016-02-05 2025-06-01 Univ Emory Administration of single stranded or self-complementary adeno-associated virus 9 by injection into the cerebrospinal fluid to target gene therapy to the central nervous system
CA3016314A1 (en) 2016-03-02 2017-09-08 Julianne REIDERS Therapy for frontotemporal dementia
US11802294B2 (en) 2017-10-03 2023-10-31 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
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
BR112020008033A2 (pt) 2017-10-23 2020-10-27 Prevail Therapeutics, Inc. terapias gênicas para doença neurodegenerativa
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
WO2020210713A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders

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