NZ704275A - Aav-mediated gene therapy for rpgr x-linked retinal degeneration - Google Patents

Aav-mediated gene therapy for rpgr x-linked retinal degeneration

Info

Publication number
NZ704275A
NZ704275A NZ704275A NZ70427513A NZ704275A NZ 704275 A NZ704275 A NZ 704275A NZ 704275 A NZ704275 A NZ 704275A NZ 70427513 A NZ70427513 A NZ 70427513A NZ 704275 A NZ704275 A NZ 704275A
Authority
NZ
New Zealand
Prior art keywords
aav
subject
rpgr
retinitis pigmentosa
gene therapy
Prior art date
Application number
NZ704275A
Other languages
English (en)
Inventor
William A Beltran
Gustavo D Aguirre
Samuel G Jacobson
Artur V Cideciyan
Alfred S Lewin
Sanford L Boye
William W Hauswirth
wen-tao Deng
Original Assignee
Univ Florida
Univ Pennsylvania
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 Florida, Univ Pennsylvania filed Critical Univ Florida
Publication of NZ704275A publication Critical patent/NZ704275A/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
    • A61K38/46—Hydrolases (3)
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • 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
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/05—Hydrolases acting on acid anhydrides (3.6) acting on GTP; involved in cellular and subcellular movement (3.6.5)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Psychology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
NZ704275A 2012-07-11 2013-01-23 Aav-mediated gene therapy for rpgr x-linked retinal degeneration NZ704275A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261670355P 2012-07-11 2012-07-11
PCT/US2013/022628 WO2014011210A1 (en) 2012-07-11 2013-01-23 Aav-mediated gene therapy for rpgr x-linked retinal degeneration

Publications (1)

Publication Number Publication Date
NZ704275A true NZ704275A (en) 2016-09-30

Family

ID=49916455

Family Applications (1)

Application Number Title Priority Date Filing Date
NZ704275A NZ704275A (en) 2012-07-11 2013-01-23 Aav-mediated gene therapy for rpgr x-linked retinal degeneration

Country Status (8)

Country Link
US (2) US9770491B2 (https=)
EP (1) EP2872183B1 (https=)
JP (1) JP6199965B2 (https=)
CN (1) CN105120901A (https=)
AU (1) AU2013287281B2 (https=)
CA (1) CA2878171C (https=)
NZ (1) NZ704275A (https=)
WO (1) WO2014011210A1 (https=)

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EP2872183B1 (en) * 2012-07-11 2018-09-26 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
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EP3748007B1 (en) * 2015-03-06 2024-06-26 Massachusetts Eye & Ear Infirmary Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene
EP3268481B1 (en) * 2015-03-11 2020-05-27 The United States of America, as represented by The Secretary, Department of Health and Human Services Rpgr vectors for treating x-linked retinitis pigmentosa
HK1253299A1 (zh) * 2015-06-04 2019-06-14 INSERM (Institut National de la Santé et de la Recherche Médicale) 一种通过基因治疗治疗眼疾病的改进方法
GB201516066D0 (en) 2015-09-10 2015-10-28 Young & Co Llp D Treatment of retinitis pigmentosa
CA3005474A1 (en) 2015-11-19 2017-05-26 The Trustees Of The University Of Pennsylvania Compositions and methods for correction of heritable ocular disease
CA3008264A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
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WO2018009814A1 (en) 2016-07-08 2018-01-11 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving rdh12
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WO2018200542A1 (en) 2017-04-24 2018-11-01 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
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KR102704296B1 (ko) * 2017-11-15 2024-09-05 더 리젠츠 오브 더 유니버시티 오브 미시간 Rdh12 암호화 영역을 포함하는 바이러스 벡터 및 망막 이영양증을 치료하는 방법
KR102866133B1 (ko) 2018-04-17 2025-09-30 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 트랜스-스플라이싱 분자
WO2020061528A1 (en) 2018-09-21 2020-03-26 University Of Connecticut Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome
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CN113795279B (zh) 2019-03-04 2024-12-31 宾夕法尼亚州大学信托人 靶向akt通路的神经保护性基因疗法
IL295673A (en) * 2020-02-28 2022-10-01 Univ Pennsylvania Treatment of autosomal dominant strophin diseases and methods of their evaluation
CN113952472A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
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WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
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Also Published As

Publication number Publication date
US20180036385A1 (en) 2018-02-08
CA2878171C (en) 2021-04-27
CN105120901A (zh) 2015-12-02
AU2013287281A1 (en) 2015-02-19
EP2872183B1 (en) 2018-09-26
AU2013287281B2 (en) 2018-04-26
US9770491B2 (en) 2017-09-26
JP6199965B2 (ja) 2017-09-20
CA2878171A1 (en) 2014-01-16
JP2015523379A (ja) 2015-08-13
US20150202269A1 (en) 2015-07-23
EP2872183A4 (en) 2015-12-09
WO2014011210A1 (en) 2014-01-16
EP2872183A1 (en) 2015-05-20
US10383922B2 (en) 2019-08-20

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