DK3191139T3 - RPGR-genterapi til retinitis pigmentosa - Google Patents

RPGR-genterapi til retinitis pigmentosa Download PDF

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Publication number
DK3191139T3
DK3191139T3 DK15825383.1T DK15825383T DK3191139T3 DK 3191139 T3 DK3191139 T3 DK 3191139T3 DK 15825383 T DK15825383 T DK 15825383T DK 3191139 T3 DK3191139 T3 DK 3191139T3
Authority
DK
Denmark
Prior art keywords
gene therapy
retinitis pigmentosa
rpgr gene
rpgr
pigmentosa
Prior art date
Application number
DK15825383.1T
Other languages
English (en)
Inventor
Michael A Sandberg
Basil Pawlyk
Tiansen Li
Xinhua Shu
Alan Finlay Wright
Robin Ali
Original Assignee
Massachusetts Eye & Ear Infirmary
The Government Of The Us Secretary Department Of Health And Human Services
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 Massachusetts Eye & Ear Infirmary, The Government Of The Us Secretary Department Of Health And Human Services, Ucl Business Ltd filed Critical Massachusetts Eye & Ear Infirmary
Application granted granted Critical
Publication of DK3191139T3 publication Critical patent/DK3191139T3/da

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Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Zoology (AREA)
  • Toxicology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Compounds Of Unknown Constitution (AREA)
  • Saccharide Compounds (AREA)
DK15825383.1T 2014-07-24 2015-07-17 RPGR-genterapi til retinitis pigmentosa DK3191139T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462028638P 2014-07-24 2014-07-24
PCT/US2015/040866 WO2016014353A1 (en) 2014-07-24 2015-07-17 Rpgr gene therapy for retinitis pigmentosa

Publications (1)

Publication Number Publication Date
DK3191139T3 true DK3191139T3 (da) 2020-11-16

Family

ID=55163577

Family Applications (1)

Application Number Title Priority Date Filing Date
DK15825383.1T DK3191139T3 (da) 2014-07-24 2015-07-17 RPGR-genterapi til retinitis pigmentosa

Country Status (16)

Country Link
US (2) US10314924B2 (da)
EP (2) EP3821912B1 (da)
JP (3) JP6654760B2 (da)
CN (1) CN107206105A (da)
CA (1) CA2991750C (da)
CY (1) CY1123793T1 (da)
DK (1) DK3191139T3 (da)
ES (1) ES2834402T3 (da)
HR (1) HRP20202023T1 (da)
HU (1) HUE052781T2 (da)
LT (1) LT3191139T (da)
PL (1) PL3191139T3 (da)
PT (1) PT3191139T (da)
RS (1) RS61307B1 (da)
SI (1) SI3191139T1 (da)
WO (1) WO2016014353A1 (da)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107206105A (zh) * 2014-07-24 2017-09-26 麻省眼耳科医院 色素性视网膜炎的rpgr基因疗法
US20190054117A1 (en) * 2014-12-19 2019-02-21 Novartis Ag Dimerization switches and uses thereof
AU2016228751B2 (en) * 2015-03-11 2022-01-13 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Rp2 vectors for treating x-linked retinitis pigmentosa
GB201516066D0 (en) 2015-09-10 2015-10-28 Young & Co Llp D Treatment of retinitis pigmentosa
GB201704192D0 (en) 2017-03-16 2017-05-03 Nightstarx Ltd Treatment of Retinitis Pigmentosa
WO2019077159A1 (en) 2017-10-20 2019-04-25 INSERM (Institut National de la Santé et de la Recherche Médicale) METHODS OF EXPRESSING POLYNUCLEOTIDE OF INTEREST IN CONE PHOTORECCEPTORS OF A SUBJECT COMPRISING SUB-RETINAL ADMINISTRATION OF A THERAPEUTICALLY EFFECTIVE AMOUNT OF A RECOMBINANT AAV9 DERIVATIVE VECTOR
CN111886340A (zh) * 2017-11-15 2020-11-03 密歇根大学董事会 包括rdh12编码区的病毒载体和治疗视网膜营养不良的方法
MX2022011177A (es) * 2020-03-11 2022-12-13 Massachusetts Eye & Ear Infirmary Terapia genica para la degeneracion de la retina asociada a nmnat1.

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE68013T1 (de) 1985-07-05 1991-10-15 Whitehead Biomedical Inst Expression von fremdem genetischem material in epithelzellen.
US4980286A (en) 1985-07-05 1990-12-25 Whitehead Institute For Biomedical Research In vivo introduction and expression of foreign genetic material in epithelial cells
JPS6259797A (ja) * 1985-09-10 1987-03-16 日本電信電話株式会社 地中管体の推進方法
ATE117375T1 (de) 1987-09-11 1995-02-15 Whitehead Biomedical Inst Transduktionsveränderte fibroblasten und ihre anwendung.
DE3851153T2 (de) 1987-12-11 1995-01-05 Hughes Howard Med Inst Genetische modifizierung von endothelialen zellen.
DE68927996T2 (de) 1988-02-05 1997-12-04 Hughes Howard Med Inst Modifizierte hepatozyten und deren anwendung
ATE163046T1 (de) 1990-10-31 1998-02-15 Somatix Therapy Corp Genetische veränderung von endothelzellen
US5767079A (en) * 1992-07-08 1998-06-16 Celtrix Pharmaceuticals, Inc. Method of treating ophthalmic disorders using TGF -β
AU2044901A (en) 1999-11-24 2001-06-04 Cornell Research Foundation Inc. Inherited retinal diseases at the canine rp3 locus: linkage, marker- and mutation-based tests
GB0008801D0 (en) 2000-04-10 2000-05-31 Medical Res Council Sequences
CN102925445A (zh) * 2011-08-08 2013-02-13 刘军 一种获得rpgr 基因新的转录剪切形式的方法
CN105120901A (zh) * 2012-07-11 2015-12-02 宾夕法尼亚大学托管会 Rpgr x染色体连锁视网膜退化的aav介导的基因治疗
GB201412011D0 (en) * 2014-07-04 2014-08-20 Ucl Business Plc Treatments
CN107206105A (zh) * 2014-07-24 2017-09-26 麻省眼耳科医院 色素性视网膜炎的rpgr基因疗法

Also Published As

Publication number Publication date
WO2016014353A1 (en) 2016-01-28
HUE052781T2 (hu) 2021-05-28
EP3821912B1 (en) 2024-06-12
HRP20202023T1 (hr) 2021-03-05
EP3191139B1 (en) 2020-10-07
CY1123793T1 (el) 2022-05-27
JP7198329B2 (ja) 2022-12-28
US10314924B2 (en) 2019-06-11
LT3191139T (lt) 2021-01-25
CA2991750C (en) 2023-02-14
US11045558B2 (en) 2021-06-29
JP2017523239A (ja) 2017-08-17
CN107206105A (zh) 2017-09-26
US20170216454A1 (en) 2017-08-03
EP3821912A1 (en) 2021-05-19
CA2991750A1 (en) 2016-01-28
EP3191139A4 (en) 2018-01-10
JP6654760B2 (ja) 2020-02-26
JP2022009333A (ja) 2022-01-14
JP2020073536A (ja) 2020-05-14
JP6966532B2 (ja) 2021-11-17
ES2834402T3 (es) 2021-06-17
RS61307B1 (sr) 2021-02-26
PL3191139T3 (pl) 2021-07-05
PT3191139T (pt) 2020-11-05
US20200215203A1 (en) 2020-07-09
SI3191139T1 (sl) 2021-03-31
EP3191139A1 (en) 2017-07-19

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