FI3710590T3 - Virusvektoreita sisältäviä RDH12:ta koodaavia alueita ja menetelmiä verkkokalvon dystrofioiden hoitoon - Google Patents

Virusvektoreita sisältäviä RDH12:ta koodaavia alueita ja menetelmiä verkkokalvon dystrofioiden hoitoon Download PDF

Info

Publication number
FI3710590T3
FI3710590T3 FIEP18878835.0T FI18878835T FI3710590T3 FI 3710590 T3 FI3710590 T3 FI 3710590T3 FI 18878835 T FI18878835 T FI 18878835T FI 3710590 T3 FI3710590 T3 FI 3710590T3
Authority
FI
Finland
Prior art keywords
adeno
human
associated virus
virus vector
rdh12
Prior art date
Application number
FIEP18878835.0T
Other languages
English (en)
Finnish (fi)
Inventor
Debra A Thompson
Robin R Ali
Alexander J Smith
Original Assignee
Univ Michigan Regents
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 filed Critical Univ Michigan Regents
Application granted granted Critical
Publication of FI3710590T3 publication Critical patent/FI3710590T3/fi

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)
  • 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)
FIEP18878835.0T 2017-11-15 2018-11-15 Virusvektoreita sisältäviä RDH12:ta koodaavia alueita ja menetelmiä verkkokalvon dystrofioiden hoitoon FI3710590T3 (fi)

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
FI3710590T3 true FI3710590T3 (fi) 2024-08-06

Family

ID=66431192

Family Applications (1)

Application Number Title Priority Date Filing Date
FIEP18878835.0T FI3710590T3 (fi) 2017-11-15 2018-11-15 Virusvektoreita sisältäviä RDH12:ta koodaavia alueita ja menetelmiä verkkokalvon dystrofioiden hoitoon

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
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
ZA202003416B (en) 2024-10-30
JP2021502978A (ja) 2021-02-04
IL274609A (en) 2020-06-30
EP4364803A3 (en) 2024-06-05

Similar Documents

Publication Publication Date Title
FI3710590T3 (fi) Virusvektoreita sisältäviä RDH12:ta koodaavia alueita ja menetelmiä verkkokalvon dystrofioiden hoitoon
JP6616319B2 (ja) 腫瘍崩壊ワクシニアウィルス
Coune et al. Parkinson’s disease: gene therapies
US20190093091A1 (en) Compositions for eradicating flavivirus infections in subjects
JP2021502978A5 (enExample)
FI3821912T3 (fi) Verkkokalvorappeuman RPGR-geeniterapia
JP2015523379A5 (enExample)
JP2017523239A5 (enExample)
JP2020513824A5 (enExample)
JP2020073536A5 (enExample)
RU2018112506A (ru) Лечение пигментного ретинита
WO2020223362A8 (en) Compositions useful for treatment of pompe disease
JP2019525744A5 (enExample)
EP2262899A1 (en) Method of treating genetic disorders
RU2019126509A (ru) Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
HK1253299A1 (zh) 一种通过基因治疗治疗眼疾病的改进方法
JP2018526994A5 (enExample)
US20230220420A1 (en) Gene therapy for bardet-biedl syndrome
WO2016001693A1 (en) Rpgr-orf15 variant gene in the treatment of retinitis pigmentosa
CN103898160A (zh) 表达光敏感型腺苷酸环化酶的重组载体、其应用、构建方法及脱髓鞘疾病的治疗系统
US12415850B2 (en) Compositions and methods for regulating production of an antibody like protein and ribonucleic acid
CN110636868A (zh) 通过适体-介导的多腺苷酸化信号可及性来调控基因表达
CN114917342B (zh) Arhgap44促进剂在抑制阿片类药物诱发的痛觉过敏中的应用
Chen et al. Recent advances in cardiac gene therapy strategies targeting advanced heart failure
CN111100847A (zh) 安全性更高的端粒基因治疗产品使人体健康逆转衰老