JP2023502515A - Ａｐｏｅ遺伝子療法 - Google Patents

Ａｐｏｅ遺伝子療法 Download PDF

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Publication number
JP2023502515A
JP2023502515A JP2022530164A JP2022530164A JP2023502515A JP 2023502515 A JP2023502515 A JP 2023502515A JP 2022530164 A JP2022530164 A JP 2022530164A JP 2022530164 A JP2022530164 A JP 2022530164A JP 2023502515 A JP2023502515 A JP 2023502515A
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vector
gene therapy
apoe4
composition
therapy vector
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JP2023502515A5 (https=
Inventor
ロナルド ジー． クリスタル
スティーブン エム． カミンスキー
ケイティ スタイルズ
ドラン ソンディ
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Cornell University
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Cornell University
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Publication of JP2023502515A5 publication Critical patent/JP2023502515A5/ja
Priority to JP2025124433A priority Critical patent/JP2025163078A/ja
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    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • 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
    • 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

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  • 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)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2022530164A 2019-11-25 2020-11-25 Ａｐｏｅ遺伝子療法 Pending JP2023502515A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025124433A JP2025163078A (ja) 2019-11-25 2025-07-25 Ａｐｏｅ遺伝子療法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962939999P 2019-11-25 2019-11-25
US62/939,999 2019-11-25
PCT/US2020/070822 WO2021108809A1 (en) 2019-11-25 2020-11-25 Apoe gene therapy

Related Child Applications (1)

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JP2025124433A Division JP2025163078A (ja) 2019-11-25 2025-07-25 Ａｐｏｅ遺伝子療法

Publications (2)

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JP2023502515A true JP2023502515A (ja) 2023-01-24
JP2023502515A5 JP2023502515A5 (https=) 2023-11-28

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JP2022530164A Pending JP2023502515A (ja) 2019-11-25 2020-11-25 Ａｐｏｅ遺伝子療法
JP2025124433A Pending JP2025163078A (ja) 2019-11-25 2025-07-25 Ａｐｏｅ遺伝子療法

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US (1) US12611467B2 (https=)
EP (1) EP4065600A1 (https=)
JP (2) JP2023502515A (https=)
KR (1) KR20220107243A (https=)
CN (1) CN114829390A (https=)
AU (1) AU2020391518B2 (https=)
BR (1) BR112022010065A2 (https=)
CA (1) CA3162761A1 (https=)
IL (1) IL293116A (https=)
MX (1) MX2022006364A (https=)
NZ (1) NZ789753A (https=)
WO (1) WO2021108809A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12611467B2 (en) 2019-11-25 2026-04-28 Cornell University APOE gene therapy

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CN119264211A (zh) 2018-08-27 2025-01-07 瑞泽恩制药公司 拉曼光谱在下游纯化中的应用
WO2020160156A2 (en) 2019-01-30 2020-08-06 Immutics, Inc. Anti-gal3 antibodies and uses thereof
EP4157338A4 (en) 2020-05-26 2024-11-13 TrueBinding, Inc. METHOD FOR TREATING INFLAMMATORY DISEASES BY GALECTIN-3 BLOCKING
KR20230112672A (ko) * 2020-11-25 2023-07-27 프리베일 테라퓨틱스, 인크. 신경변성 질환을 위한 유전자 요법
WO2023198702A1 (en) * 2022-04-12 2023-10-19 Uniqure Biopharma B.V. Nucleic acid regulation of c9orf72
JP2025513066A (ja) * 2022-04-12 2025-04-22 ユニキュアー バイオファーマ ビー．ブイ． Ａｐｏｅの核酸制御
EP4551711A1 (en) * 2022-07-08 2025-05-14 Cornell University Methods and pharmaceutical compositions for the treatment and the prevention of alzheimers disease
CN121335984A (zh) * 2023-04-18 2026-01-13 康奈尔大学 用于治疗和预防阿尔茨海默病的方法和apoe药物组合物
WO2025213111A2 (en) * 2024-04-05 2025-10-09 Cornell University Aav expression of mirna to suppress human apoe mrna

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Cited By (1)

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Publication number Priority date Publication date Assignee Title
US12611467B2 (en) 2019-11-25 2026-04-28 Cornell University APOE gene therapy

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AU2020391518B2 (en) 2025-02-13
CA3162761A1 (en) 2021-06-03
NZ789753A (en) 2026-02-27
CN114829390A (zh) 2022-07-29
MX2022006364A (es) 2022-06-22
EP4065600A1 (en) 2022-10-05
IL293116A (en) 2022-07-01
JP2025163078A (ja) 2025-10-28
AU2020391518A1 (en) 2022-07-14
US12611467B2 (en) 2026-04-28
KR20220107243A (ko) 2022-08-02
US20230044351A1 (en) 2023-02-09
WO2021108809A1 (en) 2021-06-03
BR112022010065A2 (pt) 2022-09-06

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