ZA202003416B - 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
ZA202003416B
ZA202003416B ZA2020/03416A ZA202003416A ZA202003416B ZA 202003416 B ZA202003416 B ZA 202003416B ZA 2020/03416 A ZA2020/03416 A ZA 2020/03416A ZA 202003416 A ZA202003416 A ZA 202003416A ZA 202003416 B ZA202003416 B ZA 202003416B
Authority
ZA
South Africa
Prior art keywords
methods
viral vectors
coding regions
treating retinal
retinal dystrophies
Prior art date
Application number
ZA2020/03416A
Other languages
English (en)
Inventor
A Thompson Debra
R Ali Robin
J Smith Alexander
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 ZA202003416B publication Critical patent/ZA202003416B/en

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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)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (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)
ZA2020/03416A 2017-11-15 2020-06-08 Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies ZA202003416B (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
ZA202003416B true ZA202003416B (en) 2024-10-30

Family

ID=66431192

Family Applications (1)

Application Number Title Priority Date Filing Date
ZA2020/03416A ZA202003416B (en) 2017-11-15 2020-06-08 Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies

Country Status (27)

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

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL264070B2 (en) 2016-07-08 2023-03-01 Univ Pennsylvania Methods and preparations for the treatment of disorders and diseases involving rdh12
EP4329882A4 (en) * 2021-04-27 2025-02-26 The Trustees of Columbia University in the City of New York METHODS AND COMPOSITIONS FOR REGULATING 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
AU2011242527B2 (en) * 2010-04-23 2016-05-19 University Of Florida Research Foundation, Inc. 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
NZ704275A (en) * 2012-07-11 2016-09-30 Univ Florida Aav-mediated gene therapy for rpgr x-linked retinal degeneration
PE20170260A1 (es) * 2014-05-02 2017-04-12 Genzyme Corp Vectores de aav para la terapia genica de la retina y el snc
GB201412011D0 (en) * 2014-07-04 2014-08-20 Ucl Business Plc Treatments
DK3821912T3 (da) * 2014-07-24 2024-08-05 Massachusetts Eye & Ear Infirmary RPGR-genterapi mod 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
EP4008780A1 (en) * 2015-04-30 2022-06-08 The Trustees of Columbia University in the City of New York Gene therapy for autosomal dominant diseases
IL264070B2 (en) 2016-07-08 2023-03-01 Univ Pennsylvania Methods and preparations for the treatment of disorders and diseases involving rdh12

Also Published As

Publication number Publication date
US20190142909A1 (en) 2019-05-16
SI3710590T1 (sl) 2024-10-30
IL308645A (en) 2024-01-01
EP3710590A4 (en) 2021-06-23
PL3710590T3 (pl) 2024-10-21
EA202091194A1 (ru) 2020-08-14
EP3710590B1 (en) 2024-07-17
WO2019099696A1 (en) 2019-05-23
CA3082586A1 (en) 2019-05-23
KR20200088385A (ko) 2020-07-22
SMT202400321T1 (it) 2024-11-15
MY204271A (en) 2024-08-20
CN117752822A (zh) 2024-03-26
EP4364803A2 (en) 2024-05-08
MX2020005008A (es) 2020-11-06
IL274609B1 (en) 2024-01-01
JP7615238B2 (ja) 2025-01-16
RS65881B1 (sr) 2024-09-30
KR102704296B1 (ko) 2024-09-05
IL274609B2 (en) 2024-05-01
SG11202004527YA (en) 2020-06-29
HUE068393T2 (hu) 2024-12-28
MA50914A (fr) 2020-09-23
PT3710590T (pt) 2024-08-09
AU2018367606A1 (en) 2020-06-25
EP3710590A1 (en) 2020-09-23
FI3710590T3 (fi) 2024-08-06
ES2983990T3 (es) 2024-10-28
HRP20241073T1 (hr) 2024-11-08
BR112020009828A2 (pt) 2020-11-17
US20230190884A1 (en) 2023-06-22
DK3710590T3 (da) 2024-08-12
PH12020551319A1 (en) 2021-09-01
CN111886340A (zh) 2020-11-03
LT3710590T (lt) 2024-09-25
AU2018367606B2 (en) 2024-08-29
IL308645B1 (en) 2025-09-01
JP2023133434A (ja) 2023-09-22
JP2021502978A (ja) 2021-02-04
IL274609A (en) 2020-06-30
EP4364803A3 (en) 2024-06-05

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