JP2022522004A - 依存症障害の遺伝子治療 - Google Patents

依存症障害の遺伝子治療 Download PDF

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Publication number
JP2022522004A
JP2022522004A JP2021550130A JP2021550130A JP2022522004A JP 2022522004 A JP2022522004 A JP 2022522004A JP 2021550130 A JP2021550130 A JP 2021550130A JP 2021550130 A JP2021550130 A JP 2021550130A JP 2022522004 A JP2022522004 A JP 2022522004A
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JP
Japan
Prior art keywords
rvv
vector
nucleic acid
condition
virus
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Japanese (ja)
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JP2022522004A5 (OSRAM
Inventor
ウォーレン シー． ラウ，
ブラッド トンプソン，
Original Assignee
パスウェイズ ニューロ ファーマ， インコーポレイテッド
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Publication of JP2022522004A publication Critical patent/JP2022522004A/ja
Publication of JP2022522004A5 publication Critical patent/JP2022522004A5/ja
Pending legal-status Critical Current

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    • 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
    • C07K14/70571—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • 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
    • 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
    • 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
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0356—Animal model for processes and diseases of the central nervous system, e.g. stress, learning, schizophrenia, pain, epilepsy
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021550130A 2019-02-27 2020-02-27 依存症障害の遺伝子治療 Pending JP2022522004A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962811339P 2019-02-27 2019-02-27
US62/811,339 2019-02-27
PCT/US2020/020216 WO2020176796A1 (en) 2019-02-27 2020-02-27 Gene therapy for addiction disorders

Publications (2)

Publication Number Publication Date
JP2022522004A true JP2022522004A (ja) 2022-04-13
JP2022522004A5 JP2022522004A5 (OSRAM) 2023-03-07

Family

ID=72239690

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021550130A Pending JP2022522004A (ja) 2019-02-27 2020-02-27 依存症障害の遺伝子治療

Country Status (5)

Country Link
US (1) US20220152222A1 (OSRAM)
EP (1) EP3931214A4 (OSRAM)
JP (1) JP2022522004A (OSRAM)
AU (2) AU2020229874A1 (OSRAM)
WO (1) WO2020176796A1 (OSRAM)

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018531926A (ja) * 2015-09-17 2018-11-01 コーダ バイオセラピューティクス， インコーポレイテッド 神経学的障害を処置するための組成物および方法

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU775525B2 (en) * 1999-01-22 2004-08-05 Auckland Technology Enabling Corporation Limited Vaccine-mediated treatment of neurological disorders
US20070065801A1 (en) * 2003-05-21 2007-03-22 Bayer Healthcare Ag Diagnostics and therapeutics for diseases associated with 5-hydroxytryptamine (seotonin) receptor 4 (5-ht4)
GB201404470D0 (en) * 2014-03-13 2014-04-30 Ucl Business Plc Therapeutic methods and materials

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018531926A (ja) * 2015-09-17 2018-11-01 コーダ バイオセラピューティクス， インコーポレイテッド 神経学的障害を処置するための組成物および方法

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
"UniProt", Q13639, FULL=5-HYDROXYTRYPTAMINE RECEPTOR 4, JPN6024008839, 13 February 2019 (2019-02-13), ISSN: 0005426157 *

Also Published As

Publication number Publication date
AU2020229874A1 (en) 2021-09-23
US20220152222A1 (en) 2022-05-19
WO2020176796A1 (en) 2020-09-03
AU2025248711A1 (en) 2025-11-13
EP3931214A4 (en) 2023-09-13
EP3931214A1 (en) 2022-01-05

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