SG11202004527YA - Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies - Google Patents

Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies

Info

Publication number
SG11202004527YA
SG11202004527YA SG11202004527YA SG11202004527YA SG11202004527YA SG 11202004527Y A SG11202004527Y A SG 11202004527YA SG 11202004527Y A SG11202004527Y A SG 11202004527YA SG 11202004527Y A SG11202004527Y A SG 11202004527YA SG 11202004527Y A SG11202004527Y A SG 11202004527YA
Authority
SG
Singapore
Prior art keywords
methods
viral vectors
coding regions
treating retinal
retinal dystrophies
Prior art date
Application number
SG11202004527YA
Inventor
Debra A Thompson
Robin R Ali
Alexander J Smith
Original Assignee
Univ Michigan Regents
Ucl Business Ltd
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, Ucl Business Ltd filed Critical Univ Michigan Regents
Publication of SG11202004527YA publication Critical patent/SG11202004527YA/en

Links

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)
    • 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; CARE OF BIRDS, FISHES, INSECTS; 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; CARE OF BIRDS, FISHES, INSECTS; 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; CARE OF BIRDS, FISHES, INSECTS; 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
SG11202004527YA 2017-11-15 2018-11-15 Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies SG11202004527YA (en)

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
SG11202004527YA true SG11202004527YA (en) 2020-06-29

Family

ID=66431192

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202004527YA SG11202004527YA (en) 2017-11-15 2018-11-15 Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies

Country Status (15)

Country Link
US (2) US20190142909A1 (en)
EP (1) EP3710590A4 (en)
JP (2) JP2021502978A (en)
KR (1) KR20200088385A (en)
CN (2) CN117752822A (en)
AU (1) AU2018367606A1 (en)
BR (1) BR112020009828A2 (en)
CA (1) CA3082586A1 (en)
EA (1) EA202091194A1 (en)
IL (2) IL274609B1 (en)
MA (1) MA50914A (en)
MX (1) MX2020005008A (en)
PH (1) PH12020551319A1 (en)
SG (1) SG11202004527YA (en)
WO (1) WO2019099696A1 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

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
PT2561067T (en) * 2010-04-23 2019-02-04 Univ Florida Raav-guanylate cyclase compositions and methods for treating leber's congenital amaurosis-1 (lca1)
WO2012167109A2 (en) * 2011-06-03 2012-12-06 Massachusetts Eye & Ear Infirmary Rpgrip1 gene therapy for leber congenital amaurosis
US9770491B2 (en) * 2012-07-11 2017-09-26 The Trustees Of The University Of Pennsylvania AAV-mediated gene therapy for RPGR X-linked retinal degeneration
CN114231563A (en) * 2014-05-02 2022-03-25 建新公司 AAV vectors for retinal and CNS gene therapy
GB201412011D0 (en) 2014-07-04 2014-08-20 Ucl Business Plc Treatments
HUE052781T2 (en) * 2014-07-24 2021-05-28 Massachusetts Eye & Ear Infirmary Rpgr gene therapy for retinitis pigmentosa
CA2979229A1 (en) * 2015-03-11 2016-09-15 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Rp2 vectors for treating x-linked retinitis pigmentosa
ES2898917T3 (en) * 2015-04-30 2022-03-09 Univ Columbia Gene therapy for autosomal dominant diseases
KR102526506B1 (en) 2016-07-08 2023-05-03 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Methods and compositions for the treatment of disorders and diseases involving RDH12

Also Published As

Publication number Publication date
MA50914A (en) 2020-09-23
CN117752822A (en) 2024-03-26
AU2018367606A1 (en) 2020-06-25
IL274609A (en) 2020-06-30
BR112020009828A2 (en) 2020-11-17
MX2020005008A (en) 2020-11-06
KR20200088385A (en) 2020-07-22
EA202091194A1 (en) 2020-08-14
US20190142909A1 (en) 2019-05-16
PH12020551319A1 (en) 2021-09-01
JP2021502978A (en) 2021-02-04
IL308645A (en) 2024-01-01
EP3710590A4 (en) 2021-06-23
US20230190884A1 (en) 2023-06-22
JP2023133434A (en) 2023-09-22
EP3710590A1 (en) 2020-09-23
WO2019099696A1 (en) 2019-05-23
IL274609B1 (en) 2024-01-01
CN111886340A (en) 2020-11-03
CA3082586A1 (en) 2019-05-23

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