BR112021019880A2 - Terapias gênicas para distúrbios lisossomais - Google Patents

Terapias gênicas para distúrbios lisossomais

Info

Publication number
BR112021019880A2
BR112021019880A2 BR112021019880A BR112021019880A BR112021019880A2 BR 112021019880 A2 BR112021019880 A2 BR 112021019880A2 BR 112021019880 A BR112021019880 A BR 112021019880A BR 112021019880 A BR112021019880 A BR 112021019880A BR 112021019880 A2 BR112021019880 A2 BR 112021019880A2
Authority
BR
Brazil
Prior art keywords
disease
gene therapies
parkinson
lysosomal disorders
gaucher
Prior art date
Application number
BR112021019880A
Other languages
English (en)
Inventor
Asa Abeliovich
Franz Hefti
Herve Rhinn
Laura Heckman
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 BR112021019880A2 publication Critical patent/BR112021019880A2/pt

Links

Classifications

    • 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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/162—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/20—Animals treated with compounds which are neither proteins nor nucleic acids
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Psychology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)

Abstract

terapias gênicas para distúrbios lisossomais. a presente invenção refere-se a composições e métodos para o tratamento de doenças associadas à função lisossomal aberrante, como o mal de parkinson e a doença de gaucher. a presente invenção refere-se a construtos de expressão que compreendem um transgene que codifica beta-glicocerebrosidase, um rna inibidor que direciona a alfa-sinucleína ou uma combinação dos anteriores. a divulgação fornece adicionalmente métodos de tratamento da doença de gaucher, mal de parkinson ou outras sinucleinopatias pela administração de tais construtos de expressão a um indivíduo em necessidade do mesmo.
BR112021019880A 2019-04-10 2020-04-10 Terapias gênicas para distúrbios lisossomais BR112021019880A2 (pt)

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
BR112021019880A2 true BR112021019880A2 (pt) 2022-02-15

Family

ID=70554182

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021019880A BR112021019880A2 (pt) 2019-04-10 2020-04-10 Terapias gênicas para distúrbios lisossomais

Country Status (11)

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

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2020004005A (es) 2017-10-03 2020-10-05 Prevail Therapeutics Inc Terapias genicas para los trastornos lisosomales.
WO2019070893A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISORDERS
KR20200074132A (ko) 2017-10-03 2020-06-24 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
MX2021011958A (es) 2019-04-10 2021-12-15 Prevail Therapeutics Inc Terapias genicas para trastornos lisosomales.
CA3190866A1 (en) * 2020-08-10 2022-02-17 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
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
WO1998009524A1 (en) 1996-09-06 1998-03-12 Chiron Corporation Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors
ATE454445T1 (de) 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
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
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
ES2252293T3 (es) 2000-09-18 2006-05-16 Genzyme Corporation Vectores de expresion que contienene promotores hibridos de ubiquitina.
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
WO2006039253A2 (en) 2004-09-29 2006-04-13 Children's Memorial Hospital Sirna-mediated gene silencing of alpha synuclein
JP2009509913A (ja) * 2005-06-08 2009-03-12 アミカス セラピューティックス インコーポレイテッド リソソーム酵素をコードする遺伝子の変異に関連するｃｎｓ障害の治療
DK2061891T3 (da) 2006-08-24 2012-07-23 Virovek Inc Ekspression i insektceller af gener med overlappende åbne læserammer, fremgangsmåder og sammensætninger hertil
WO2008143797A1 (en) 2007-05-11 2008-11-27 Genzyme Corporation Methods of producing a secreted protein
EP3000320B1 (en) 2007-05-16 2019-01-16 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
WO2010129791A1 (en) 2009-05-06 2010-11-11 University Of Medicine And Dentistry Of New Jersey Rna targeting in alpha-synucleinopathies
CA3049237C (en) 2010-04-23 2024-06-11 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
CN103189507A (zh) 2010-10-27 2013-07-03 学校法人自治医科大学 用于向神经***细胞导入基因的腺相关病毒粒子
WO2012065248A1 (en) 2010-11-16 2012-05-24 Kay Denis G Method for increasing neprilysin expression and activity
EP2707369B1 (en) 2011-05-09 2016-07-27 Eip Pharma, LLC Compositions and methods for treating alzheimer'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
KR20240093939A (ko) 2013-05-15 2024-06-24 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
US9347107B2 (en) 2013-05-23 2016-05-24 Norman Z Lai Vector containing multiple nucleotide sequences for the expression of enzymes
WO2015060722A1 (en) 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
PT3137497T (pt) * 2014-05-02 2021-07-12 Genzyme Corp Vetores de aav para terapia genética na retina e snc
EP3221456B1 (en) 2014-11-21 2021-09-22 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
CN108367055A (zh) 2015-05-15 2018-08-03 明尼苏达大学董事会 用于治疗性递送到中枢神经***的腺相关物
CA3002406A1 (en) 2015-10-29 2017-05-04 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
EP3370778A1 (en) 2015-11-05 2018-09-12 Bamboo Therapeutics, Inc. Modified friedreich ataxia genes and vectors for gene therapy
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
PL3411484T3 (pl) 2016-02-05 2024-02-19 Emory University Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego
EP3423109A4 (en) 2016-03-02 2019-08-14 The Children's Hospital of Philadelphia THERAPY FOR FRONTOTEMPORAL DEMENTIA
KR20230079511A (ko) 2016-04-21 2023-06-07 바이로베크 인코포레이티드 곤충 세포에서의 aav 생산, 및 이를 위한 방법 및 조성물
KR20200074132A (ko) 2017-10-03 2020-06-24 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
WO2019070893A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISORDERS
MX2020004005A (es) 2017-10-03 2020-10-05 Prevail Therapeutics Inc Terapias genicas para los trastornos lisosomales.
WO2019084068A1 (en) 2017-10-23 2019-05-02 Prevail Therapeutics, Inc. GENE THERAPIES FOR NEURODEGENERATIVE DISEASE
AU2020272974A1 (en) 2019-04-10 2021-11-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
US20220211871A1 (en) 2019-04-10 2022-07-07 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders

Also Published As

Publication number Publication date
AU2020270984A1 (en) 2021-12-02
CA3136117A1 (en) 2020-10-15
JP2022526021A (ja) 2022-05-20
US11903985B2 (en) 2024-02-20
KR20220006527A (ko) 2022-01-17
MX2021012184A (es) 2022-01-24
IL286901A (en) 2021-10-31
EP3953378A1 (en) 2022-02-16
US20200338148A1 (en) 2020-10-29
CN114026115A (zh) 2022-02-08
WO2020210713A1 (en) 2020-10-15
US20240123003A1 (en) 2024-04-18

Similar Documents

Publication Publication Date Title
BR112021019880A2 (pt) Terapias gênicas para distúrbios lisossomais
MX2020004005A (es) Terapias genicas para los trastornos lisosomales.
MX2020003965A (es) Terapias genicas para los trastornos lisosomales.
MX2023010958A (es) Agentes de arn modificados con efecto reducido fuera de la diana.
BR112021020157A2 (pt) Terapias gênicas para transtornos lisossomais
NZ767118A (en) Modified double-stranded rna agents
BR112014011896A2 (pt) agentes de rnai, composições e métodos de uso destes para o tratamento de doenças associadas à transtirretina (ttr)
MX2020003557A (es) Terapias genicas para los trastornos lisosomales.
BR112021020183A2 (pt) Terapias gênicas para distúrbios lisossomais
AR044007A1 (es) Metodos para el tratamiento de la enfermedad de parkinson
UY30097A1 (es) Usos terapeuticos de inhibidores de rtp801
BR112022002442A2 (pt) Terapias de imuno-oncologia com conjugados de il-2
EA200901166A1 (ru) Соединения и композиции в качестве модуляторов активности gpr 119
MX2009006339A (es) Compuestos y composiciones como inhibidores de actividad de receptor 1 de cannabinoide.
CL2021001490A1 (es) Constructos de iarn modificados químicamente y usos de estos
BR112018013084A2 (pt) tratamentos de combinação compreendendo a administração de imidazopirazinonas
BR112015017775A2 (pt) enzima cistationina beta-sintase para o tratamento de homocistinúria
WO2019217397A3 (en) Compositions and methods for improving strand biased
BR112015022577A2 (pt) moduladores ship1 e métodos relacionados a esse
MX2022011550A (es) Composiciones y métodos para inhibir la expresión de angptl3.
BR112014031788A2 (pt) composições e métodos para o tratamento de diabetes
BR112013020586A2 (pt) Uso de células derivadas de tecido do cordão umbilical
MY149171A (en) Treatment and prevention of influenza
MX2021006253A (es) Terapias génicas para enfermedad neurodegenerativa.
BR112021015751A2 (pt) Vetores de terapia genética para tratamento da doença de danon