JP2021502978A - 網膜ジストロフィーを治療するｒｄｈ１２コード領域を含むウイルスベクターおよび方法 - Google Patents

網膜ジストロフィーを治療するｒｄｈ１２コード領域を含むウイルスベクターおよび方法 Download PDF

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JP2021502978A
JP2021502978A JP2020526473A JP2020526473A JP2021502978A JP 2021502978 A JP2021502978 A JP 2021502978A JP 2020526473 A JP2020526473 A JP 2020526473A JP 2020526473 A JP2020526473 A JP 2020526473A JP 2021502978 A JP2021502978 A JP 2021502978A
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rdh12
human
nucleic acid
retinal
protein
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JP2021502978A5 (enExample
Inventor
エー． トンプソン、デブラ
エー． トンプソン、デブラ
アール． アリ、ロビン
アール． アリ、ロビン
ジェー． スミス、アレクサンダー
ジェー． スミス、アレクサンダー
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UCL Business Ltd
University of Michigan System
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UCL Business Ltd
University of Michigan System
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Publication of JP2021502978A publication Critical patent/JP2021502978A/ja
Publication of JP2021502978A5 publication Critical patent/JP2021502978A5/ja
Priority to JP2023121924A priority Critical patent/JP7615238B2/ja
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K38/44—Oxidoreductases (1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K38/44—Oxidoreductases (1)
    • A61K38/443—Oxidoreductases (1) acting on CH-OH groups as donors, e.g. glucose oxidase, lactate dehydrogenase (1.1)
    • 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
    • A61K48/005—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
    • C12Y101/01—Oxidoreductases acting on the CH-OH group of donors (1.1) with NAD+ or NADP+ as acceptor (1.1.1)
    • C12Y101/01105—Retinol dehydrogenase (1.1.1.105)
    • 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
    • A01K2217/00—Genetically modified animals
    • 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
    • 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
    • A01K2267/0306—Animal model for genetic diseases
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043—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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • 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)
JP2020526473A 2017-11-15 2018-11-15 網膜ジストロフィーを治療するｒｄｈ１２コード領域を含むウイルスベクターおよび方法 Pending JP2021502978A (ja)

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JP2023121924A JP7615238B2 (ja) 2017-11-15 2023-07-26 網膜ジストロフィーを治療するｒｄｈ１２コード領域を含むウイルスベクターおよび方法

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US201762586624P 2017-11-15 2017-11-15
US62/586,624 2017-11-15
PCT/US2018/061319 WO2019099696A1 (en) 2017-11-15 2018-11-15 Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies

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JP2023121924A Active JP7615238B2 (ja) 2017-11-15 2023-07-26 網膜ジストロフィーを治療するｒｄｈ１２コード領域を含むウイルスベクターおよび方法

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Publication number Priority date Publication date Assignee Title
KR102526506B1 (ko) 2016-07-08 2023-05-03 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물
WO2022232249A1 (en) * 2021-04-27 2022-11-03 The Trustees Of Columbia University In The City Of New York Methods and compositions to regulate cholesterol efflux to prevent, treat, or cure macular degeneration
CN115725659A (zh) * 2022-04-01 2023-03-03 首都医科大学附属北京同仁医院 一种应用视网膜原位基因编辑制作非人灵长类动物先天性黑矇模型的方法
CN118453915A (zh) * 2024-04-29 2024-08-09 中国医学科学院北京协和医院 一种针对rdh12突变的基因治疗药物
CN119432856B (zh) * 2024-12-30 2025-08-12 云舟生物科技(广州)股份有限公司 核酸分子、病毒颗粒及其制备方法和应用

Citations (4)

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WO2011034947A2 (en) * 2009-09-15 2011-03-24 University Of Washington Reagents and methods for modulating cone photoreceptor activity
WO2012167109A2 (en) * 2011-06-03 2012-12-06 Massachusetts Eye & Ear Infirmary Rpgrip1 gene therapy for leber congenital amaurosis
JP2013526854A (ja) * 2010-04-23 2013-06-27 ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド レーバー先天性黒内障１（ＬＣＡ１）を処置するためのｒＡＡＶ−グアニル酸シクラーゼ組成物および方法
JP2017518271A (ja) * 2014-05-02 2017-07-06 ジェンザイム・コーポレーション 網膜およびｃｎｓ遺伝子治療用ａａｖベクター

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GB201412011D0 (en) 2014-07-04 2014-08-20 Ucl Business Plc Treatments
HRP20241154T1 (hr) * 2014-07-24 2024-11-22 Massachusetts Eye & Ear Infirmary Rpgr genska terapija za pigmentnu retinopatiju
AU2016228751B2 (en) * 2015-03-11 2022-01-13 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Rp2 vectors for treating x-linked retinitis pigmentosa
WO2016176690A2 (en) * 2015-04-30 2016-11-03 The Trustees Of Columbia University In The City Of New York Gene therapy for autosomal dominant diseases
KR102526506B1 (ko) 2016-07-08 2023-05-03 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물

Patent Citations (4)

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WO2011034947A2 (en) * 2009-09-15 2011-03-24 University Of Washington Reagents and methods for modulating cone photoreceptor activity
JP2013526854A (ja) * 2010-04-23 2013-06-27 ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド レーバー先天性黒内障１（ＬＣＡ１）を処置するためのｒＡＡＶ−グアニル酸シクラーゼ組成物および方法
WO2012167109A2 (en) * 2011-06-03 2012-12-06 Massachusetts Eye & Ear Infirmary Rpgrip1 gene therapy for leber congenital amaurosis
JP2017518271A (ja) * 2014-05-02 2017-07-06 ジェンザイム・コーポレーション 網膜およびｃｎｓ遺伝子治療用ａａｖベクター

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LT3710590T (lt) 2024-09-25
MA50914A (fr) 2020-09-23
AU2018367606B2 (en) 2024-08-29
HUE068393T2 (hu) 2024-12-28
KR102704296B1 (ko) 2024-09-05
ES2983990T3 (es) 2024-10-28
BR112020009828A2 (pt) 2020-11-17
IL274609A (en) 2020-06-30
CA3082586A1 (en) 2019-05-23
ZA202003416B (en) 2024-10-30
EP3710590A1 (en) 2020-09-23
US20190142909A1 (en) 2019-05-16
IL274609B1 (en) 2024-01-01
EA202091194A1 (ru) 2020-08-14
KR20200088385A (ko) 2020-07-22
MX2020005008A (es) 2020-11-06
JP2023133434A (ja) 2023-09-22
IL308645B1 (en) 2025-09-01
SMT202400321T1 (it) 2024-11-15
SG11202004527YA (en) 2020-06-29
US20230190884A1 (en) 2023-06-22
IL274609B2 (en) 2024-05-01
IL308645A (en) 2024-01-01
MY204271A (en) 2024-08-20
CN111886340A (zh) 2020-11-03
RS65881B1 (sr) 2024-09-30
SI3710590T1 (sl) 2024-10-30
PL3710590T3 (pl) 2024-10-21
EP3710590B1 (en) 2024-07-17
FI3710590T3 (fi) 2024-08-06
CN117752822A (zh) 2024-03-26
DK3710590T3 (da) 2024-08-12
AU2018367606A1 (en) 2020-06-25
EP4364803A2 (en) 2024-05-08
JP7615238B2 (ja) 2025-01-16
EP3710590A4 (en) 2021-06-23
WO2019099696A1 (en) 2019-05-23
HRP20241073T1 (hr) 2024-11-08
EP4364803A3 (en) 2024-06-05
PH12020551319A1 (en) 2021-09-01
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