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PT3821912T - Terapia do gene rpgr para retinite pigmentosa - Google Patents

Terapia do gene rpgr para retinite pigmentosa

Info

Publication number
PT3821912T
PT3821912T PT201992088T PT20199208T PT3821912T PT 3821912 T PT3821912 T PT 3821912T PT 201992088 T PT201992088 T PT 201992088T PT 20199208 T PT20199208 T PT 20199208T PT 3821912 T PT3821912 T PT 3821912T
Authority
PT
Portugal
Prior art keywords
gene therapy
retinitis pigmentosa
rpgr gene
rpgr
pigmentosa
Prior art date
Application number
PT201992088T
Other languages
English (en)
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
Publication of PT3821912T publication Critical patent/PT3821912T/pt

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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • 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
    • 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
    • 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)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Ophthalmology & Optometry (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Zoology (AREA)
  • Toxicology (AREA)
  • Biotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Bioinformatics & Cheminformatics (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)
  • Saccharide Compounds (AREA)
  • Compounds Of Unknown Constitution (AREA)
PT201992088T 2014-07-24 2015-07-17 Terapia do gene rpgr para retinite pigmentosa PT3821912T (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201462028638P 2014-07-24 2014-07-24

Publications (1)

Publication Number Publication Date
PT3821912T true PT3821912T (pt) 2024-08-01

Family

ID=55163577

Family Applications (2)

Application Number Title Priority Date Filing Date
PT158253831T PT3191139T (pt) 2014-07-24 2015-07-17 Terapia do gene rpgr para retinite pigmentosa
PT201992088T PT3821912T (pt) 2014-07-24 2015-07-17 Terapia do gene rpgr para retinite pigmentosa

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PT158253831T PT3191139T (pt) 2014-07-24 2015-07-17 Terapia do gene rpgr para retinite pigmentosa

Country Status (18)

Country Link
US (2) US10314924B2 (pt)
EP (2) EP3821912B1 (pt)
JP (3) JP6654760B2 (pt)
CN (1) CN107206105A (pt)
CA (1) CA2991750C (pt)
CY (1) CY1123793T1 (pt)
DK (2) DK3191139T3 (pt)
ES (2) ES2987090T3 (pt)
FI (1) FI3821912T3 (pt)
HR (2) HRP20241154T1 (pt)
HU (2) HUE068183T2 (pt)
LT (2) LT3191139T (pt)
PL (2) PL3191139T3 (pt)
PT (2) PT3191139T (pt)
RS (2) RS61307B1 (pt)
SI (2) SI3191139T1 (pt)
SM (2) SMT202000715T1 (pt)
WO (1) WO2016014353A1 (pt)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016014353A1 (en) 2014-07-24 2016-01-28 Massachusetts Eye & Ear Infirmary Rpgr gene therapy for retinitis pigmentosa
US20190054117A1 (en) * 2014-12-19 2019-02-21 Novartis Ag Dimerization switches and uses thereof
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
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
EP4364803A3 (en) * 2017-11-15 2024-06-05 The Regents of the University of Michigan Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies
JP2023517929A (ja) * 2020-03-11 2023-04-27 マサチューセッツ アイ アンド イヤー インファーマリー Ｎｍｎａｔ１関連網膜変性に対する遺伝子療法

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU6131086A (en) 1985-07-05 1987-01-30 Whitehead Institute For Biomedical Research Epithelial cells expressing foreign genetic material
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
JP3015383B2 (ja) 1987-09-11 2000-03-06 ホワイトヘツド・インスチチユート・フオー・バイオメデイカル・リサーチ 形質導入した線維芽およびそれらの使用
EP0391960B1 (en) 1987-12-11 1994-08-17 Whitehead Institute For Biomedical Research Genetic modification of endothelial cells
DE68927996T2 (de) 1988-02-05 1997-12-04 Hughes Howard Med Inst Modifizierte hepatozyten und deren anwendung
AU656544B2 (en) 1990-10-31 1995-02-09 Brigham And Women's Hospital Genetic modification of endothelial cells
US5767079A (en) * 1992-07-08 1998-06-16 Celtrix Pharmaceuticals, Inc. Method of treating ophthalmic disorders using TGF -β
WO2001038578A1 (en) * 1999-11-24 2001-05-31 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
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
GB201412011D0 (en) * 2014-07-04 2014-08-20 Ucl Business Plc Treatments
WO2016014353A1 (en) * 2014-07-24 2016-01-28 Massachusetts Eye & Ear Infirmary Rpgr gene therapy for retinitis pigmentosa

Also Published As

Publication number Publication date
CA2991750A1 (en) 2016-01-28
PL3821912T3 (pl) 2024-10-28
HUE052781T2 (hu) 2021-05-28
FI3821912T3 (fi) 2024-08-05
US20170216454A1 (en) 2017-08-03
US10314924B2 (en) 2019-06-11
US20200215203A1 (en) 2020-07-09
JP2022009333A (ja) 2022-01-14
LT3191139T (lt) 2021-01-25
SI3191139T1 (sl) 2021-03-31
EP3821912A1 (en) 2021-05-19
SI3821912T1 (sl) 2024-10-30
CN107206105A (zh) 2017-09-26
PL3191139T3 (pl) 2021-07-05
LT3821912T (lt) 2024-09-25
HRP20241154T1 (hr) 2024-11-22
DK3821912T3 (da) 2024-08-05
EP3191139A4 (en) 2018-01-10
EP3191139A1 (en) 2017-07-19
RS65909B1 (sr) 2024-10-31
JP2020073536A (ja) 2020-05-14
JP6654760B2 (ja) 2020-02-26
RS61307B1 (sr) 2021-02-26
SMT202000715T1 (it) 2021-01-05
EP3191139B1 (en) 2020-10-07
PT3191139T (pt) 2020-11-05
HRP20202023T1 (hr) 2021-03-05
HUE068183T2 (hu) 2024-12-28
WO2016014353A1 (en) 2016-01-28
EP3821912B1 (en) 2024-06-12
CY1123793T1 (el) 2022-05-27
US11045558B2 (en) 2021-06-29
CA2991750C (en) 2023-02-14
JP6966532B2 (ja) 2021-11-17
ES2834402T3 (es) 2021-06-17
JP2017523239A (ja) 2017-08-17
ES2987090T3 (es) 2024-11-13
SMT202400309T1 (it) 2024-11-15
JP7198329B2 (ja) 2022-12-28
DK3191139T3 (da) 2020-11-16

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