EA202091194A1 - Вирусные векторы, содержащие области, кодирующие rdh12, и способы лечения дистрофий сетчатки - Google Patents

Вирусные векторы, содержащие области, кодирующие rdh12, и способы лечения дистрофий сетчатки

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Publication number
EA202091194A1
EA202091194A1 EA202091194A EA202091194A EA202091194A1 EA 202091194 A1 EA202091194 A1 EA 202091194A1 EA 202091194 A EA202091194 A EA 202091194A EA 202091194 A EA202091194 A EA 202091194A EA 202091194 A1 EA202091194 A1 EA 202091194A1
Authority
EA
Eurasian Patent Office
Prior art keywords
treatment
methods
viral vectors
vectors containing
containing regions
Prior art date
Application number
EA202091194A
Other languages
English (en)
Inventor
Дебра А. Томпсон
Робин Р. Али
Александер Дж. Смит
Original Assignee
Дзе Риджентс Оф Дзе Юниверсити Оф Мичиган
ЮСиЭл БИЗНЕС ЛТД
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.)
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Application filed by Дзе Риджентс Оф Дзе Юниверсити Оф Мичиган, ЮСиЭл БИЗНЕС ЛТД filed Critical Дзе Риджентс Оф Дзе Юниверсити Оф Мичиган
Publication of EA202091194A1 publication Critical patent/EA202091194A1/ru

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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)
    • 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

Abstract

Изобретение относится к материалам, способам и применениям для лечения офтальмологической патологии, такой как врожденный амавроз Лебера, путем введения эффективного количества адено-ассоциированного вируса AAV2, серотипа 5 (AAV 2/5) или AAV-5, содержащих пригодную для экспрессии кодирующую область для RDH12 человека.
EA202091194A 2017-11-15 2018-11-15 Вирусные векторы, содержащие области, кодирующие rdh12, и способы лечения дистрофий сетчатки EA202091194A1 (ru)

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
EA202091194A1 true EA202091194A1 (ru) 2020-08-14

Family

ID=66431192

Family Applications (1)

Application Number Title Priority Date Filing Date
EA202091194A EA202091194A1 (ru) 2017-11-15 2018-11-15 Вирусные векторы, содержащие области, кодирующие rdh12, и способы лечения дистрофий сетчатки

Country Status (15)

Country Link
US (2) US20190142909A1 (ru)
EP (2) EP4364803A2 (ru)
JP (2) JP2021502978A (ru)
KR (1) KR20200088385A (ru)
CN (2) CN111886340A (ru)
AU (1) AU2018367606A1 (ru)
BR (1) BR112020009828A2 (ru)
CA (1) CA3082586A1 (ru)
EA (1) EA202091194A1 (ru)
IL (2) IL308645A (ru)
MA (1) MA50914A (ru)
MX (1) MX2020005008A (ru)
PH (1) PH12020551319A1 (ru)
SG (1) SG11202004527YA (ru)
WO (1) WO2019099696A1 (ru)

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
WO2011034947A2 (en) * 2009-09-15 2011-03-24 University Of Washington Reagents and methods for modulating cone photoreceptor activity
EA202091105A1 (ru) * 2010-04-23 2020-12-30 Юниверсити Оф Флорида Рисерч Фаундейшн, Инк. Композиции гуанилатциклазы и способы лечения врожденного амавроза лебера типа 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
SG10201809739QA (en) * 2014-05-02 2018-12-28 Genzyme Corp Aav vectors for retinal and cns gene therapy
GB201412011D0 (en) 2014-07-04 2014-08-20 Ucl Business Plc Treatments
ES2834402T3 (es) * 2014-07-24 2021-06-17 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
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
CN110191954A (zh) 2016-07-08 2019-08-30 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
IL274609A (en) 2020-06-30
MX2020005008A (es) 2020-11-06
IL274609B1 (en) 2024-01-01
EP3710590A1 (en) 2020-09-23
BR112020009828A2 (pt) 2020-11-17
CN111886340A (zh) 2020-11-03
MA50914A (fr) 2020-09-23
WO2019099696A1 (en) 2019-05-23
JP2023133434A (ja) 2023-09-22
US20230190884A1 (en) 2023-06-22
US20190142909A1 (en) 2019-05-16
EP3710590A4 (en) 2021-06-23
SG11202004527YA (en) 2020-06-29
AU2018367606A1 (en) 2020-06-25
CA3082586A1 (en) 2019-05-23
KR20200088385A (ko) 2020-07-22
CN117752822A (zh) 2024-03-26
IL308645A (en) 2024-01-01
EP4364803A2 (en) 2024-05-08
PH12020551319A1 (en) 2021-09-01
JP2021502978A (ja) 2021-02-04

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