IL308645B1 - Viral vectors comprising RDH12 coding regions and methods for treating retinal dystrophies - Google Patents

Viral vectors comprising RDH12 coding regions and methods for treating retinal dystrophies

Info

Publication number
IL308645B1
IL308645B1 IL308645A IL30864523A IL308645B1 IL 308645 B1 IL308645 B1 IL 308645B1 IL 308645 A IL308645 A IL 308645A IL 30864523 A IL30864523 A IL 30864523A IL 308645 B1 IL308645 B1 IL 308645B1
Authority
IL
Israel
Prior art keywords
adeno
viral vector
associated viral
use according
seq
Prior art date
Application number
IL308645A
Other languages
English (en)
Hebrew (he)
Other versions
IL308645A (en
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 IL308645A publication Critical patent/IL308645A/en
Publication of IL308645B1 publication Critical patent/IL308645B1/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)
    • 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)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (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)
IL308645A 2017-11-15 2018-11-15 Viral vectors comprising RDH12 coding regions and methods for treating retinal dystrophies IL308645B1 (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 (2)

Publication Number Publication Date
IL308645A IL308645A (en) 2024-01-01
IL308645B1 true IL308645B1 (en) 2025-09-01

Family

ID=66431192

Family Applications (2)

Application Number Title Priority Date Filing Date
IL308645A IL308645B1 (en) 2017-11-15 2018-11-15 Viral vectors comprising RDH12 coding regions and methods for treating retinal dystrophies
IL274609A IL274609B2 (en) 2017-11-15 2018-11-15 Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies

Family Applications After (1)

Application Number Title Priority Date Filing Date
IL274609A IL274609B2 (en) 2017-11-15 2018-11-15 Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies

Country Status (27)

Country Link
US (2) US20190142909A1 (enExample)
EP (2) EP3710590B1 (enExample)
JP (2) JP2021502978A (enExample)
KR (1) KR102704296B1 (enExample)
CN (2) CN111886340A (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) IL308645B1 (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
CN110191954B (zh) 2016-07-08 2024-05-14 宾夕法尼亚州大学信托人 用于治疗涉及rdh12的病症和疾病的方法和组合物
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
JP6199965B2 (ja) * 2012-07-11 2017-09-20 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 Ｒｐｇｒｘ連鎖性網膜変性のａａｖ媒介型遺伝子治療
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
GB201412011D0 (en) * 2014-07-04 2014-08-20 Ucl Business Plc Treatments
ES2987090T3 (es) * 2014-07-24 2024-11-13 Massachusetts Eye & Ear Infirmary Terapia génica con RPGR para 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
PT3289080T (pt) * 2015-04-30 2021-11-19 Univ Columbia Terapia genética para doenças autossómicas dominantes
CN110191954B (zh) * 2016-07-08 2024-05-14 宾夕法尼亚州大学信托人 用于治疗涉及rdh12的病症和疾病的方法和组合物

Also Published As

Publication number Publication date
JP7615238B2 (ja) 2025-01-16
EA202091194A1 (ru) 2020-08-14
LT3710590T (lt) 2024-09-25
EP3710590A1 (en) 2020-09-23
CN111886340A (zh) 2020-11-03
DK3710590T3 (da) 2024-08-12
SG11202004527YA (en) 2020-06-29
SMT202400321T1 (it) 2024-11-15
EP4364803A3 (en) 2024-06-05
JP2023133434A (ja) 2023-09-22
US20190142909A1 (en) 2019-05-16
WO2019099696A1 (en) 2019-05-23
MA50914A (fr) 2020-09-23
US20230190884A1 (en) 2023-06-22
CN117752822A (zh) 2024-03-26
RS65881B1 (sr) 2024-09-30
PH12020551319A1 (en) 2021-09-01
AU2018367606B2 (en) 2024-08-29
SI3710590T1 (sl) 2024-10-30
PL3710590T3 (pl) 2024-10-21
PT3710590T (pt) 2024-08-09
EP3710590A4 (en) 2021-06-23
KR20200088385A (ko) 2020-07-22
IL274609A (en) 2020-06-30
ZA202003416B (en) 2024-10-30
MX2020005008A (es) 2020-11-06
CA3082586A1 (en) 2019-05-23
EP4364803A2 (en) 2024-05-08
ES2983990T3 (es) 2024-10-28
IL274609B2 (en) 2024-05-01
BR112020009828A2 (pt) 2020-11-17
EP3710590B1 (en) 2024-07-17
KR102704296B1 (ko) 2024-09-05
AU2018367606A1 (en) 2020-06-25
IL308645A (en) 2024-01-01
JP2021502978A (ja) 2021-02-04
FI3710590T3 (fi) 2024-08-06
HRP20241073T1 (hr) 2024-11-08
HUE068393T2 (hu) 2024-12-28
MY204271A (en) 2024-08-20
IL274609B1 (en) 2024-01-01

Similar Documents

Publication Publication Date Title
IL308645B1 (en) Viral vectors comprising RDH12 coding regions and methods for treating retinal dystrophies
PH12020550706A1 (en) Adeno-associated virus variant capsids and use for inhibiting angiogenesis
JP2015523379A5 (enExample)
JP2017523239A5 (enExample)
JP2020073536A5 (enExample)
JP2018520646A5 (enExample)
FI3821912T3 (fi) Verkkokalvorappeuman RPGR-geeniterapia
FI4342992T3 (fi) Geenihoito neurodegeneratiivisiin sairauksiin
RU2019100525A (ru) Векторная система на основе аденоассоциированного вируса
JP2015508760A5 (enExample)
FI3795181T3 (fi) Amd:n hoito aav2-variantilla ja afliberseptillä
WO2015150914A3 (en) Systemic delivery of virus vectors encoding urocortin-2 and related genes to treat diabetes-related cardiac dysfunction and congestive heart failure
PH12021552638A1 (en) Compositions useful for treatment of pompe disease
RU2018112506A (ru) Лечение пигментного ретинита
HRP20201225T1 (hr) Terapije poboljšavanja gena kod nasljedne degeneracije mrežnice uzrokovanu mutacijama u genu prpf31
FI4017871T3 (fi) Alfa-sarkoglykaaniin liittyvä adeno-assosioidun virusvektorin antaminen ja lihasdystrofian hoito
JP2021507687A5 (enExample)
JP2021502978A5 (enExample)
RU2018114907A (ru) Композиция и способ для лечения заболевания, опосредованного комплементом
HK1253299A1 (zh) 一种通过基因治疗治疗眼疾病的改进方法
WO2022229703A3 (en) New aav8 based immune escaping variants
JP2017533910A5 (enExample)
JP2015514115A5 (enExample)
RU2019126509A (ru) Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
JP2018509154A5 (enExample)