IL273776B2 - Gene therapies for lysosomal disorders - Google Patents

Gene therapies for lysosomal disorders

Info

Publication number
IL273776B2
IL273776B2 IL273776A IL27377620A IL273776B2 IL 273776 B2 IL273776 B2 IL 273776B2 IL 273776 A IL273776 A IL 273776A IL 27377620 A IL27377620 A IL 27377620A IL 273776 B2 IL273776 B2 IL 273776B2
Authority
IL
Israel
Prior art keywords
itr
raav
sequence
expression construct
vector
Prior art date
Application number
IL273776A
Other languages
English (en)
Hebrew (he)
Other versions
IL273776A (en
IL273776B1 (en
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 IL273776A publication Critical patent/IL273776A/en
Publication of IL273776B1 publication Critical patent/IL273776B1/en
Publication of IL273776B2 publication Critical patent/IL273776B2/en

Links

Classifications

    • 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
    • C12N15/861—Adenoviral vectors
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • C07K14/475—Growth factors; Growth regulators
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70596—Molecules with a "CD"-designation not provided for elsewhere
    • 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
    • C12N15/11—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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Psychology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • 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)
IL273776A 2017-10-03 2018-10-03 Gene therapies for lysosomal disorders IL273776B2 (en)

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 (3)

Publication Number Publication Date
IL273776A IL273776A (en) 2020-05-31
IL273776B1 IL273776B1 (en) 2025-02-01
IL273776B2 true IL273776B2 (en) 2025-06-01

Family

ID=65994357

Family Applications (2)

Application Number Title Priority Date Filing Date
IL278868A IL278868B2 (en) 2017-10-03 2018-10-03 Gene therapies for lysosomal disorders
IL273776A IL273776B2 (en) 2017-10-03 2018-10-03 Gene therapies for lysosomal disorders

Family Applications Before (1)

Application Number Title Priority Date Filing Date
IL278868A IL278868B2 (en) 2017-10-03 2018-10-03 Gene therapies for lysosomal disorders

Country Status (11)

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

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20200078513A (ko) 2017-10-03 2020-07-01 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
JP7361037B2 (ja) 2017-10-03 2023-10-13 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
US11802294B2 (en) * 2017-10-03 2023-10-31 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CA3128875A1 (en) 2019-02-04 2020-08-13 Freeline Therapeutics Limited Polynucleotides
BR112021019880A2 (pt) 2019-04-10 2022-02-15 Prevail Therapeutics Inc Terapias gênicas para distúrbios lisossomais
CN114174324A (zh) 2019-04-10 2022-03-11 普利维尔治疗公司 用于溶酶体病症的基因疗法
CA3149844A1 (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
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
US20230257436A1 (en) * 2020-06-18 2023-08-17 Alan F. Feeney Esq. Cell Lines Secreting Alpha-Synuclein Targeting Antibodies, Progranulin and Prosaposin and a Complex of Both, and GDNF
KR20230093241A (ko) 2020-07-27 2023-06-27 보이저 테라퓨틱스, 인크. 글루코실세라미데이스 베타 결핍증과 관련된 신경 장애의 치료를 위한 조성물 및 방법
BR112023001583A2 (pt) 2020-07-29 2023-02-14 Freeline Therapeutics Ltd Polipeptídeos de ss-glucocerebrosidase (gcase) modificado, polinucleotídeo, partícula viral e composição
CN116157527A (zh) * 2020-08-10 2023-05-23 普利维尔治疗公司 用于溶酶体病症的基因疗法
US20230346979A1 (en) 2020-08-10 2023-11-02 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disorders
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
WO2022081985A1 (en) 2020-10-15 2022-04-21 Prevail Therapeutics, Inc. Assay for measuring potency of gene therapy drug product
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
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
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

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002024932A2 (en) * 2000-09-18 2002-03-28 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
WO2014071282A1 (en) * 2012-11-05 2014-05-08 Genzyme Corporation Compositions and methods for treating proteinopathies
WO2016081927A2 (en) * 2014-11-21 2016-05-26 University Of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
WO2017077451A1 (en) * 2015-11-05 2017-05-11 Bamboo Therapeutics, Inc. Modified friedreich ataxia genes and vectors for gene therapy

Family Cites Families (38)

* 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
US6491907B1 (en) 1998-11-10 2002-12-10 The University Of North Carolina At Chapel Hill Recombinant parvovirus vectors and method of making
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
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
DE602005015994D1 (de) 2004-09-29 2009-09-24 Childrens Memorial Hospital siRNA-VERMITTELTES GEN-SILENCING VON ALPHA-SYNUKLEIN
CA2654292C (en) 2006-06-07 2022-01-11 Genzyme Corporation Gene therapy for motor neuron disorders
US8962273B2 (en) 2007-05-11 2015-02-24 Genzyme Corporation Methods of producing a secreted protein
PL2154969T3 (pl) 2007-05-16 2016-04-29 Brigham & Womens Hospital Inc Leczenie synukleinopatii
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
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
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
CA2863829A1 (en) * 2012-02-19 2013-08-22 Protalix Ltd. Oral unit dosage forms and uses of same for the treatment of gaucher disease
EP3622821A1 (en) 2013-05-15 2020-03-18 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
US9574184B2 (en) 2013-09-25 2017-02-21 Children's Hospital Medical Center Lysosomal protein targeting sequence and therapeutic applications of same
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
SMT202200022T1 (it) 2015-05-15 2022-03-21 Regenxbio Inc Virus adeno-associato per veicolazione terapeutica al sistema nervoso centrale
MX2018004755A (es) 2015-10-29 2018-12-19 Voyager Therapeutics Inc Entrega de polinucleotidos dirigidos al sistema nervioso central.
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
SI3411484T1 (sl) 2016-02-05 2023-12-29 Emory University Injiciranje enojnega ali samokomplementarnega adeno-povezanega virusa 9 v cerebrospinalno tekočino
CA3016314A1 (en) 2016-03-02 2017-09-08 Julianne REIDERS Therapy for frontotemporal dementia
KR20200078513A (ko) 2017-10-03 2020-07-01 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
US11802294B2 (en) 2017-10-03 2023-10-31 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
JP7361037B2 (ja) 2017-10-03 2023-10-13 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
US20210261981A1 (en) 2017-10-23 2021-08-26 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative diseases
US20220211871A1 (en) 2019-04-10 2022-07-07 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
BR112021020157A2 (pt) 2019-04-10 2021-12-14 Prevail Therapeutics Inc Terapias gênicas para transtornos lisossomais
BR112021019880A2 (pt) 2019-04-10 2022-02-15 Prevail Therapeutics Inc Terapias gênicas para distúrbios lisossomais

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002024932A2 (en) * 2000-09-18 2002-03-28 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
WO2014071282A1 (en) * 2012-11-05 2014-05-08 Genzyme Corporation Compositions and methods for treating proteinopathies
WO2016081927A2 (en) * 2014-11-21 2016-05-26 University Of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
WO2017077451A1 (en) * 2015-11-05 2017-05-11 Bamboo Therapeutics, Inc. Modified friedreich ataxia genes and vectors for gene therapy

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
SINCLAIR, GRAHAM, AND FRANCIS YM CHOY., SYNONYMOUS CODON USAGE BIAS AND THE EXPRESSION OF HUMAN GLUCOCEREBROSIDASE IN THE METHYLOTROPHIC YEAST, PICHIA PASTORIS., 31 December 2002 (2002-12-31) *

Also Published As

Publication number Publication date
US11060113B2 (en) 2021-07-13
US20200071726A1 (en) 2020-03-05
IL278868B1 (en) 2025-02-01
CN111542549A (zh) 2020-08-14
JP7254815B2 (ja) 2023-04-10
IL278868A (en) 2021-01-31
JP2025063034A (ja) 2025-04-15
AU2020205228B2 (en) 2023-09-07
US11802294B2 (en) 2023-10-31
US10837028B2 (en) 2020-11-17
WO2019070893A1 (en) 2019-04-11
JP2020537543A (ja) 2020-12-24
IL278868B2 (en) 2025-06-01
US20210010032A1 (en) 2021-01-14
US20210332385A1 (en) 2021-10-28
IL273776A (en) 2020-05-31
JP2023086740A (ja) 2023-06-22
MX2020011745A (es) 2021-01-08
BR112020006633A2 (pt) 2020-10-06
AU2018346104A1 (en) 2020-04-30
AU2020205228A1 (en) 2020-07-30
KR20200078512A (ko) 2020-07-01
KR102693318B1 (ko) 2024-08-07
EP3692075A1 (en) 2020-08-12
EP3692075A4 (en) 2022-02-09
AU2018346104B2 (en) 2023-07-27
IL273776B1 (en) 2025-02-01
CA3078371A1 (en) 2019-04-11
US20200231954A1 (en) 2020-07-23
MX2020003557A (es) 2020-10-07

Similar Documents

Publication Publication Date Title
IL273776B2 (en) Gene therapies for lysosomal disorders
US12297447B2 (en) Gene therapy for juvenile Batten disease
JP2020537544A5 (OSRAM)
US20210261981A1 (en) Gene therapies for neurodegenerative diseases
US10016514B2 (en) Polynucleotides, vectors and methods for insertion and expression of transgenes
US20230270884A1 (en) Compositions useful for treatment of charcot-marie-tooth disease
US20240318199A1 (en) Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegenerative diseases or disorders
US20230044220A1 (en) Treatment of chronic pain
US20200283800A1 (en) Gene therapies for neurodegenerative diseases
CN120548368A (zh) 用于正确包装重复元件的腺相关病毒载体
CN116096904A (zh) 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途
JP2023526923A (ja) ポンペ病の治療に有用な組成物
JP2023519762A (ja) ジスフェリン二重ベクターを用いた遺伝子治療
US20240401078A1 (en) Compositions useful for treatment of charcot-marie-tooth disease
CN120210188A (zh) 用于调控基因表达的杂合启动子及其用途
CN120173938A (zh) 用于调控基因表达的pgk-5ui系列启动子及其用途
JP2024515612A (ja) 球脊髄性筋萎縮症（ｓｂｍａ）の治療に有用な組成物
CN121195072A (zh) 用于帕金森病的基因治疗载体