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DK3710590T3 - Virale vektorer omfattende rdh12-kodende områder og fremgangsmåder til behandling af nethindedystrofier - Google Patents

Virale vektorer omfattende rdh12-kodende områder og fremgangsmåder til behandling af nethindedystrofier Download PDF

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Publication number
DK3710590T3
DK3710590T3 DK18878835.0T DK18878835T DK3710590T3 DK 3710590 T3 DK3710590 T3 DK 3710590T3 DK 18878835 T DK18878835 T DK 18878835T DK 3710590 T3 DK3710590 T3 DK 3710590T3
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DK
Denmark
Prior art keywords
treatment
methods
viral vectors
coding regions
retinal dystrophies
Prior art date
Application number
DK18878835.0T
Other languages
English (en)
Inventor
Debra A Thompson
Robin R Ali
Alexander J Smith
Original Assignee
Univ Michigan Regents
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
Application filed by Univ Michigan Regents filed Critical Univ Michigan Regents
Application granted granted Critical
Publication of DK3710590T3 publication Critical patent/DK3710590T3/da

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Classifications

    • 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

Landscapes

  • 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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (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)
DK18878835.0T 2017-11-15 2018-11-15 Virale vektorer omfattende rdh12-kodende områder og fremgangsmåder til behandling af nethindedystrofier DK3710590T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762586624P 2017-11-15 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

Publications (1)

Publication Number Publication Date
DK3710590T3 true DK3710590T3 (da) 2024-08-12

Family

ID=66431192

Family Applications (1)

Application Number Title Priority Date Filing Date
DK18878835.0T DK3710590T3 (da) 2017-11-15 2018-11-15 Virale vektorer omfattende rdh12-kodende områder og fremgangsmåder til behandling af nethindedystrofier

Country Status (28)

Country Link
US (2) US20190142909A1 (da)
EP (2) EP4364803A3 (da)
JP (2) JP2021502978A (da)
KR (1) KR102704296B1 (da)
CN (2) CN117752822A (da)
AU (1) AU2018367606B2 (da)
BR (1) BR112020009828A2 (da)
CA (1) CA3082586A1 (da)
DK (1) DK3710590T3 (da)
EA (1) EA202091194A1 (da)
ES (1) ES2983990T3 (da)
FI (1) FI3710590T3 (da)
HR (1) HRP20241073T1 (da)
HU (1) HUE068393T2 (da)
IL (2) IL274609B2 (da)
LT (1) LT3710590T (da)
MA (1) MA50914A (da)
MX (1) MX2020005008A (da)
MY (1) MY204271A (da)
PH (1) PH12020551319A1 (da)
PL (1) PL3710590T3 (da)
PT (1) PT3710590T (da)
RS (1) RS65881B1 (da)
SG (1) SG11202004527YA (da)
SI (1) SI3710590T1 (da)
SM (1) SMT202400321T1 (da)
WO (1) WO2019099696A1 (da)
ZA (1) ZA202003416B (da)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018009814A1 (en) 2016-07-08 2018-01-11 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving rdh12
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

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120172419A1 (en) * 2009-09-15 2012-07-05 Medical College Of Wisconsin Research Foundation Inc. Reagents and methods for modulating cone photoreceptor activity
PT3486320T (pt) * 2010-04-23 2022-03-30 Univ Florida Composições de ciclase raav-guanilato e métodos para tratar a amaurose congénita 1 de leber (acl1)
WO2012167109A2 (en) * 2011-06-03 2012-12-06 Massachusetts Eye & Ear Infirmary Rpgrip1 gene therapy for leber congenital amaurosis
AU2013287281B2 (en) * 2012-07-11 2018-04-26 The Trustees Of The University Of Pennsylvania AAV-mediated gene therapy for RPGR x-linked retinal degeneration
PT3137497T (pt) * 2014-05-02 2021-07-12 Genzyme Corp Vetores de aav para terapia genética na retina e snc
GB201412011D0 (en) 2014-07-04 2014-08-20 Ucl Business Plc Treatments
SMT202400309T1 (it) * 2014-07-24 2024-11-15 Massachusetts Eye & Ear Infirmary Terapia genica rpgr per la retinite pigmentosa
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
US20190275168A1 (en) * 2015-04-30 2019-09-12 The Trustees Of Columbia University In The City Of New York Gene therapy for autosomal dominant diseases
WO2018009814A1 (en) 2016-07-08 2018-01-11 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving rdh12

Also Published As

Publication number Publication date
EA202091194A1 (ru) 2020-08-14
MX2020005008A (es) 2020-11-06
PT3710590T (pt) 2024-08-09
FI3710590T3 (fi) 2024-08-06
IL274609A (en) 2020-06-30
EP3710590A1 (en) 2020-09-23
IL308645B1 (en) 2025-09-01
US20190142909A1 (en) 2019-05-16
CA3082586A1 (en) 2019-05-23
ZA202003416B (en) 2024-10-30
KR102704296B1 (ko) 2024-09-05
EP3710590B1 (en) 2024-07-17
IL308645A (en) 2024-01-01
RS65881B1 (sr) 2024-09-30
MY204271A (en) 2024-08-20
JP2021502978A (ja) 2021-02-04
ES2983990T3 (es) 2024-10-28
JP7615238B2 (ja) 2025-01-16
PL3710590T3 (pl) 2024-10-21
BR112020009828A2 (pt) 2020-11-17
IL274609B2 (en) 2024-05-01
SG11202004527YA (en) 2020-06-29
HRP20241073T1 (hr) 2024-11-08
CN117752822A (zh) 2024-03-26
PH12020551319A1 (en) 2021-09-01
US20230190884A1 (en) 2023-06-22
CN111886340A (zh) 2020-11-03
LT3710590T (lt) 2024-09-25
EP3710590A4 (en) 2021-06-23
EP4364803A3 (en) 2024-06-05
HUE068393T2 (hu) 2024-12-28
AU2018367606A1 (en) 2020-06-25
SMT202400321T1 (it) 2024-11-15
KR20200088385A (ko) 2020-07-22
EP4364803A2 (en) 2024-05-08
MA50914A (fr) 2020-09-23
SI3710590T1 (sl) 2024-10-30
WO2019099696A1 (en) 2019-05-23
JP2023133434A (ja) 2023-09-22
AU2018367606B2 (en) 2024-08-29
IL274609B1 (en) 2024-01-01

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