IL274609B2 - 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
IL274609B2
IL274609B2 IL274609A IL27460920A IL274609B2 IL 274609 B2 IL274609 B2 IL 274609B2 IL 274609 A IL274609 A IL 274609A IL 27460920 A IL27460920 A IL 27460920A IL 274609 B2 IL274609 B2 IL 274609B2
Authority
IL
Israel
Prior art keywords
adeno
viral vector
associated viral
human
seq
Prior art date
Application number
IL274609A
Other languages
English (en)
Hebrew (he)
Other versions
IL274609A (en
IL274609B1 (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 IL274609A publication Critical patent/IL274609A/en
Publication of IL274609B1 publication Critical patent/IL274609B1/en
Publication of IL274609B2 publication Critical patent/IL274609B2/en

Links

Classifications

    • 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
    • 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
    • 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)
IL274609A 2017-11-15 2018-11-15 Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies IL274609B2 (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 (3)

Publication Number Publication Date
IL274609A IL274609A (en) 2020-06-30
IL274609B1 IL274609B1 (en) 2024-01-01
IL274609B2 true IL274609B2 (en) 2024-05-01

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 Before (1)

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

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

Citations (4)

* 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
WO2012167109A2 (en) * 2011-06-03 2012-12-06 Massachusetts Eye & Ear Infirmary Rpgrip1 gene therapy for leber congenital amaurosis
WO2015168666A2 (en) * 2014-05-02 2015-11-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
WO2016001693A1 (en) * 2014-07-04 2016-01-07 Ucl Business Plc Rpgr-orf15 variant gene in the treatment of retinitis pigmentosa

Family Cites Families (6)

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

Patent Citations (4)

* 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
WO2012167109A2 (en) * 2011-06-03 2012-12-06 Massachusetts Eye & Ear Infirmary Rpgrip1 gene therapy for leber congenital amaurosis
WO2015168666A2 (en) * 2014-05-02 2015-11-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
WO2016001693A1 (en) * 2014-07-04 2016-01-07 Ucl Business Plc Rpgr-orf15 variant gene in the treatment of retinitis pigmentosa

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
DEBRA A. THOMPSON; LIN JIA; JINGYU YAO; KECIA L. FEATHERS; SELINA A. AZAM; ALEXANDER J. SMITH; ROBIN R. ALI:, AAV-MEDIATED EXPRESSION OF HUMAN RDH12 IN MOUSE RETINA, 31 March 2012 (2012-03-31) *

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
IL308645B1 (en) 2025-09-01
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
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

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