JP2025521111A - 異染性白質ジストロフィーを治療する方法 - Google Patents

異染性白質ジストロフィーを治療する方法 Download PDF

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JP2025521111A
JP2025521111A JP2024568194A JP2024568194A JP2025521111A JP 2025521111 A JP2025521111 A JP 2025521111A JP 2024568194 A JP2024568194 A JP 2024568194A JP 2024568194 A JP2024568194 A JP 2024568194A JP 2025521111 A JP2025521111 A JP 2025521111A
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arsa
raav
seq
aav
sequence
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JP2025521111A5 (cg-RX-API-DMAC10.html
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シィアム・ラマチャンドラン
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Genzyme Corp
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Genzyme Corp
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
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    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
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    • A61K35/76—Viruses; Subviral particles; Bacteriophages
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    • 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
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    • A—HUMAN NECESSITIES
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    • 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
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    • 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
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/86—Viral vectors
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    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06001—Arylsulfatase (3.1.6.1)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
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    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
    • C12N2750/14145—Special targeting system for viral vectors
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2024568194A 2022-05-16 2023-05-15 異染性白質ジストロフィーを治療する方法 Pending JP2025521111A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263342590P 2022-05-16 2022-05-16
US63/342,590 2022-05-16
US202363459564P 2023-04-14 2023-04-14
US63/459,564 2023-04-14
PCT/US2023/067013 WO2023225481A1 (en) 2022-05-16 2023-05-15 Methods of treating metachromatic leukodystrophy

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JP2025521111A true JP2025521111A (ja) 2025-07-08
JP2025521111A5 JP2025521111A5 (cg-RX-API-DMAC10.html) 2026-05-22

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US (1) US20230398192A1 (cg-RX-API-DMAC10.html)
EP (1) EP4526439A1 (cg-RX-API-DMAC10.html)
JP (1) JP2025521111A (cg-RX-API-DMAC10.html)
KR (1) KR20250011933A (cg-RX-API-DMAC10.html)
CN (1) CN119213120A (cg-RX-API-DMAC10.html)
AU (1) AU2023273809A1 (cg-RX-API-DMAC10.html)
CA (1) CA3253612A1 (cg-RX-API-DMAC10.html)
CO (1) CO2024016901A2 (cg-RX-API-DMAC10.html)
IL (1) IL316984A (cg-RX-API-DMAC10.html)
MX (1) MX2024014114A (cg-RX-API-DMAC10.html)
TW (1) TW202403049A (cg-RX-API-DMAC10.html)
WO (1) WO2023225481A1 (cg-RX-API-DMAC10.html)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN122206466A (zh) * 2023-11-15 2026-06-12 建新公司 治疗神经变性障碍的方法
WO2026142957A1 (en) * 2024-12-24 2026-07-02 Genzyme Corporation Aav genomes encoding engineered human frataxin transgene

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6596535B1 (en) 1999-08-09 2003-07-22 Targeted Genetics Corporation Metabolically activated recombinant viral vectors and methods for the preparation and use
US7465583B2 (en) 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
US7510872B2 (en) 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
JP4559429B2 (ja) 2003-05-21 2010-10-06 ジェンザイム・コーポレーション 空キャプシドを実質的に含まない組換えａａｖビリオン調製物を生成するための方法
US7765583B2 (en) 2005-02-28 2010-07-27 France Telecom System and method for managing virtual user domains
JP5956331B2 (ja) 2009-06-16 2016-07-27 ジェンザイム・コーポレーション 組換えａａｖベクターの改良された精製方法
JP7715640B2 (ja) * 2019-05-03 2025-07-30 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 異染性白質ジストロフィーの治療に有用な組成物
PE20220233A1 (es) * 2019-06-10 2022-02-07 Homology Medicines Inc Composiciones de virus adenoasociados para la transferencia del gen de arsa y metodos de uso de las mismas
CN115023242B (zh) 2019-11-22 2025-03-14 费城儿童医院 腺相关病毒载体变体

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Publication number Publication date
KR20250011933A (ko) 2025-01-22
MX2024014114A (es) 2024-12-06
WO2023225481A1 (en) 2023-11-23
AU2023273809A1 (en) 2025-01-02
CO2024016901A2 (es) 2024-12-19
US20230398192A1 (en) 2023-12-14
EP4526439A1 (en) 2025-03-26
CA3253612A1 (en) 2023-11-23
IL316984A (en) 2025-01-01
CN119213120A (zh) 2024-12-27
TW202403049A (zh) 2024-01-16

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