WO2020112802A1 - Gene therapies for neurodegenerative disease - Google Patents

Gene therapies for neurodegenerative disease Download PDF

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Publication number
WO2020112802A1
WO2020112802A1 PCT/US2019/063289 US2019063289W WO2020112802A1 WO 2020112802 A1 WO2020112802 A1 WO 2020112802A1 US 2019063289 W US2019063289 W US 2019063289W WO 2020112802 A1 WO2020112802 A1 WO 2020112802A1
Authority
WO
WIPO (PCT)
Prior art keywords
nucleic acid
isolated nucleic
vector
apoe4
seq
Prior art date
Legal status (The legal status 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 status listed.)
Ceased
Application number
PCT/US2019/063289
Other languages
English (en)
French (fr)
Inventor
Asa Abeliovich
Laura Heckman
Herve Rhinn
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Prevail Therapeutics Inc
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
Priority to JP2021530800A priority Critical patent/JP7616995B2/ja
Priority to MX2021006253A priority patent/MX2021006253A/es
Priority to AU2019388975A priority patent/AU2019388975A1/en
Priority to EP19889757.1A priority patent/EP3887396A4/en
Priority to IL283496A priority patent/IL283496B2/en
Priority to CN201980086952.0A priority patent/CN113557243A/zh
Priority to KR1020217019516A priority patent/KR20210096168A/ko
Priority to BR112021010234-5A priority patent/BR112021010234A2/pt
Application filed by Prevail Therapeutics Inc filed Critical Prevail Therapeutics Inc
Priority to US17/297,492 priority patent/US20220010001A1/en
Priority to CA3121211A priority patent/CA3121211A1/en
Publication of WO2020112802A1 publication Critical patent/WO2020112802A1/en
Anticipated expiration legal-status Critical
Priority to JP2024128634A priority patent/JP2024177670A/ja
Ceased legal-status Critical Current

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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

Definitions

  • An isolated nucleic acid may be DNA or RNA.
  • the disclosure provides an isolated nucleic acid comprising an expression construct encoding an inhibitory nucleic acid targeting APOE4 and/or a transgene encoding an APOE2 protein or a portion thereof.
  • a composition e.g., a composition comprising an isolated nucleic acid or a vector or a rAAV as described by the disclosure is administered both peripherally and directly to the CNS of a subject.
  • a subject is administered a composition by intra-arterial injection (e.g., injection into the carotid artery) and by
  • Isolated nucleic acids encoding an APOE2-encoding transgene are utilized overexpress APOE2.
  • the APOE2 transgene is codon-optimized to differ sufficiently from the endogenous APOE2 sequence in cells such that it would not be recognized by shRNAs targeting wild-type APOE, regardless of isoform.
  • Endpoints measured are levels of amyloid-b plaques, Tau tangles, motor and cognitive endpoints, and levels of APOE4 and APOE2 proteins.
  • Clinical trials of rAAVs of the present disclosure for the prevention or treatment of AD in carriers of the APOE4 allele are performed using a study design similar to that described in Grabowski et al. (1995) Ann. Intern. Med. 122(l):33-39.
  • the rAAVs are delivered into the CSF, intraparenchymally to the hippocampus or to another brain region, or peripherally.

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)
PCT/US2019/063289 2018-11-28 2019-11-26 Gene therapies for neurodegenerative disease Ceased WO2020112802A1 (en)

Priority Applications (11)

Application Number Priority Date Filing Date Title
KR1020217019516A KR20210096168A (ko) 2018-11-28 2019-11-26 신경퇴행성 질환을 위한 유전자 요법
AU2019388975A AU2019388975A1 (en) 2018-11-28 2019-11-26 Gene therapies for neurodegenerative disease
EP19889757.1A EP3887396A4 (en) 2018-11-28 2019-11-26 GENE THERAPIES FOR NEURODEGENERATIVE DISEASES
IL283496A IL283496B2 (en) 2018-11-28 2019-11-26 Gene therapies for neurodegenerative diseases
CN201980086952.0A CN113557243A (zh) 2018-11-28 2019-11-26 用于神经变性疾病的基因疗法
JP2021530800A JP7616995B2 (ja) 2018-11-28 2019-11-26 神経変性疾患のための遺伝子治療
CA3121211A CA3121211A1 (en) 2018-11-28 2019-11-26 Gene therapies for neurodegenerative disease
BR112021010234-5A BR112021010234A2 (pt) 2018-11-28 2019-11-26 Terapias de gene para doença neurodegenerativa
US17/297,492 US20220010001A1 (en) 2018-11-28 2019-11-26 Gene therapies for neurodegenerative disease
MX2021006253A MX2021006253A (es) 2018-11-28 2019-11-26 Terapias génicas para enfermedad neurodegenerativa.
JP2024128634A JP2024177670A (ja) 2018-11-28 2024-08-05 神経変性疾患のための遺伝子治療

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862772230P 2018-11-28 2018-11-28
US62/772,230 2018-11-28

Publications (1)

Publication Number Publication Date
WO2020112802A1 true WO2020112802A1 (en) 2020-06-04

Family

ID=70852200

Family Applications (1)

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

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=)

Cited By (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021076941A1 (en) * 2019-10-16 2021-04-22 Cornell University Gene therapy for alzheimer's disease
WO2022115535A1 (en) * 2020-11-25 2022-06-02 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
WO2023078883A1 (en) * 2021-11-03 2023-05-11 F. Hoffmann-La Roche Ag Oligonucleotides for modulating apolipoprotein e4 expression
WO2024102961A1 (en) 2022-11-11 2024-05-16 Eli Lilly And Company Synthetic nucleic acids including astrocyte-directed promoter constructs and methods of using the same
US11993790B2 (en) 2017-10-03 2024-05-28 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
US12049626B2 (en) 2017-10-03 2024-07-30 Prevail Therapeutics, Inc. Gene therapy for neurodegenerative disorders
EP4232572A4 (en) * 2020-10-22 2024-09-25 Duke University COMPOSITIONS AND PROCEDURES RELATED TO ALZHEIMER’S DISEASE
US12611467B2 (en) 2020-11-25 2026-04-28 Cornell University APOE gene therapy

Families Citing this family (1)

* Cited by examiner, † Cited by third party
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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

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US20110076233A1 (en) * 2004-02-13 2011-03-31 Immunomedics, Inc. Delivery System for Cytotoxic Drugs by Bispecific Antibody Pretargeting
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Cited By (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11993790B2 (en) 2017-10-03 2024-05-28 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
US12049626B2 (en) 2017-10-03 2024-07-30 Prevail Therapeutics, Inc. Gene therapy for neurodegenerative disorders
WO2021076941A1 (en) * 2019-10-16 2021-04-22 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
WO2022115535A1 (en) * 2020-11-25 2022-06-02 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
JP2023551254A (ja) * 2020-11-25 2023-12-07 プリベイル セラピューティクス，インコーポレーテッド 神経変性疾患のための遺伝子治療
EP4251276A4 (en) * 2020-11-25 2024-10-23 Prevail Therapeutics, Inc. GENE THERAPIES FOR NEURODEGENERATIVE DISEASE
US12611467B2 (en) 2020-11-25 2026-04-28 Cornell University APOE gene therapy
WO2023078883A1 (en) * 2021-11-03 2023-05-11 F. Hoffmann-La Roche Ag Oligonucleotides for modulating apolipoprotein e4 expression
WO2024102961A1 (en) 2022-11-11 2024-05-16 Eli Lilly And Company Synthetic nucleic acids including astrocyte-directed promoter constructs and methods of using the same
TWI883636B (zh) * 2022-11-11 2025-05-11 美商美國禮來大藥廠 含有星狀細胞導向啟動子構築體之合成核酸及其使用方法

Also Published As

Publication number Publication date
US20220010001A1 (en) 2022-01-13
CA3121211A1 (en) 2020-06-04
IL283496B1 (en) 2025-05-01
IL283496B2 (en) 2025-09-01
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
EP3887396A1 (en) 2021-10-06

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