IL283496B2 - Gene therapies for neurodegenerative diseases - Google Patents

Gene therapies for neurodegenerative diseases

Info

Publication number
IL283496B2
IL283496B2 IL283496A IL28349621A IL283496B2 IL 283496 B2 IL283496 B2 IL 283496B2 IL 283496 A IL283496 A IL 283496A IL 28349621 A IL28349621 A IL 28349621A IL 283496 B2 IL283496 B2 IL 283496B2
Authority
IL
Israel
Prior art keywords
nucleic acid
isolated nucleic
vector
seq
apoe4
Prior art date
Application number
IL283496A
Other languages
English (en)
Hebrew (he)
Other versions
IL283496B1 (en
IL283496A (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 IL283496A publication Critical patent/IL283496A/en
Publication of IL283496B1 publication Critical patent/IL283496B1/en
Publication of IL283496B2 publication Critical patent/IL283496B2/en

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
    • 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
    • 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
    • C07K14/775—Apolipopeptides
    • 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
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hospice & Palliative Care (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
IL283496A 2018-11-28 2019-11-26 Gene therapies for neurodegenerative diseases IL283496B2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862772230P 2018-11-28 2018-11-28
PCT/US2019/063289 WO2020112802A1 (en) 2018-11-28 2019-11-26 Gene therapies for neurodegenerative disease

Publications (3)

Publication Number Publication Date
IL283496A IL283496A (en) 2021-07-29
IL283496B1 IL283496B1 (en) 2025-05-01
IL283496B2 true IL283496B2 (en) 2025-09-01

Family

ID=70852200

Family Applications (1)

Application Number Title Priority Date Filing Date
IL283496A IL283496B2 (en) 2018-11-28 2019-11-26 Gene therapies for neurodegenerative diseases

Country Status (11)

Country Link
US (1) US20220010001A1 (https=)
EP (1) EP3887396A4 (https=)
JP (2) JP7616995B2 (https=)
KR (1) KR20210096168A (https=)
CN (1) CN113557243A (https=)
AU (1) AU2019388975A1 (https=)
BR (1) BR112021010234A2 (https=)
CA (1) CA3121211A1 (https=)
IL (1) IL283496B2 (https=)
MX (1) MX2021006253A (https=)
WO (1) WO2020112802A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112020006671A2 (pt) 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
EP4045653A1 (en) * 2019-10-16 2022-08-24 Cornell University Gene therapy for alzheimer's disease
EP4232572A4 (en) * 2020-10-22 2024-09-25 Duke University COMPOSITIONS AND PROCEDURES RELATED TO ALZHEIMER’S DISEASE
KR20230112672A (ko) * 2020-11-25 2023-07-27 프리베일 테라퓨틱스, 인크. 신경변성 질환을 위한 유전자 요법
EP4426833A1 (en) * 2021-11-03 2024-09-11 F. Hoffmann-La Roche AG Oligonucleotides for modulating apolipoprotein e4 expression
EP4615981A1 (en) 2022-11-11 2025-09-17 Eli Lilly and Company Synthetic nucleic acids including astrocyte-directed promoter constructs and methods of using the same
CN119220609B (zh) * 2024-09-27 2025-09-12 科辉智药(深圳)新药研究中心有限公司 一种共表达TRIM11和ApoE2的核酸序列构建体及其应用

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050019927A1 (en) * 2003-07-13 2005-01-27 Markus Hildinger DECREASING GENE EXPRESSION IN A MAMMALIAN SUBJECT IN VIVO VIA AAV-MEDIATED RNAi EXPRESSION CASSETTE TRANSFER
US20070248599A1 (en) * 2006-02-21 2007-10-25 Jordan Tang Treatment of alzheimer's disease with inhibitors of apoe binding to apoe receptor
US20110076233A1 (en) * 2004-02-13 2011-03-31 Immunomedics, Inc. Delivery System for Cytotoxic Drugs by Bispecific Antibody Pretargeting
WO2015153760A2 (en) * 2014-04-01 2015-10-08 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a nervous system disorder
US20150337030A1 (en) * 2012-05-31 2015-11-26 The Trustees Of Columbia University In The City Of New York Methods to treat alzheimer's disease using apoe inhibitors

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3265577B2 (ja) * 1992-10-13 2002-03-11 デューク・ユニバーシティ アポリポ蛋白ｅの４型イソ型の測定法
AU2001253233B2 (en) * 2000-04-06 2006-12-07 Kos Pharmaceuticals, Inc. Compounds and methods for lowering cholesterol levels without inducing hypertriglyceridemia
JP6469000B2 (ja) * 2012-05-18 2019-02-13 ユニバーシティー オブ アイオワ リサーチ ファウンデーション アミロイドの沈着を処置するための方法および組成物
EP3626309B1 (en) * 2012-08-13 2023-03-08 The Rockefeller University Lxrbeta agonist for the treatment of cancer
MX2016006584A (es) * 2013-11-20 2016-09-06 Univ Iowa Res Found Métodos y composiciones para tratar los depósitos de amiloide.
EP3842514A1 (en) * 2014-09-12 2021-06-30 Whitehead Institute for Biomedical Research Cells expressing apolipoprotein e and uses thereof
WO2018045347A1 (en) * 2016-09-02 2018-03-08 Spark Therapeutics, Inc. Methods and vectors for treating cns disorders
KR102719222B1 (ko) * 2017-05-11 2024-10-21 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 신경 세로이드 지질갈색소증에 대한 유전자 요법
EP4045653A1 (en) * 2019-10-16 2022-08-24 Cornell University Gene therapy for alzheimer's disease

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050019927A1 (en) * 2003-07-13 2005-01-27 Markus Hildinger DECREASING GENE EXPRESSION IN A MAMMALIAN SUBJECT IN VIVO VIA AAV-MEDIATED RNAi EXPRESSION CASSETTE TRANSFER
US20110076233A1 (en) * 2004-02-13 2011-03-31 Immunomedics, Inc. Delivery System for Cytotoxic Drugs by Bispecific Antibody Pretargeting
US20070248599A1 (en) * 2006-02-21 2007-10-25 Jordan Tang Treatment of alzheimer's disease with inhibitors of apoe binding to apoe receptor
US20150337030A1 (en) * 2012-05-31 2015-11-26 The Trustees Of Columbia University In The City Of New York Methods to treat alzheimer's disease using apoe inhibitors
WO2015153760A2 (en) * 2014-04-01 2015-10-08 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a nervous system disorder

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
GUIMAS ALMEIDA, CLAUDIA, ET AL., IMPACT OF LATE-ONSET ALZHEIMER’S GENETIC RISK FACTORS ON BETA-AMYLOID ENDOCYTIC PRODUCTION., 31 December 2018 (2018-12-31) *

Also Published As

Publication number Publication date
US20220010001A1 (en) 2022-01-13
CA3121211A1 (en) 2020-06-04
IL283496B1 (en) 2025-05-01
JP7616995B2 (ja) 2025-01-17
MX2021006253A (es) 2021-09-21
JP2024177670A (ja) 2024-12-20
KR20210096168A (ko) 2021-08-04
BR112021010234A2 (pt) 2021-08-24
AU2019388975A1 (en) 2021-06-24
EP3887396A4 (en) 2022-09-07
IL283496A (en) 2021-07-29
JP2022511453A (ja) 2022-01-31
CN113557243A (zh) 2021-10-26
WO2020112802A1 (en) 2020-06-04
EP3887396A1 (en) 2021-10-06

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