PL3821912T3 - Terapia genowa rpgr w leczeniu retinopatii barwnikowej - Google Patents

Terapia genowa rpgr w leczeniu retinopatii barwnikowej

Info

Publication number
PL3821912T3
PL3821912T3 PL20199208.8T PL20199208T PL3821912T3 PL 3821912 T3 PL3821912 T3 PL 3821912T3 PL 20199208 T PL20199208 T PL 20199208T PL 3821912 T3 PL3821912 T3 PL 3821912T3
Authority
PL
Poland
Prior art keywords
gene therapy
retinitis pigmentosa
rpgr gene
rpgr
pigmentosa
Prior art date
Application number
PL20199208.8T
Other languages
English (en)
Polish (pl)
Inventor
Michael A SANDBERG
Basil PAWLYK
Tiansen Li
Xinhua SHU
Alan Finlay WRIGHT
Robin Ali
Original Assignee
Massachusetts Eye & Ear Infirmary
Ucl Business Ltd
The Government Of The United States Of America As Represented By The Secretary, Department Of Health And Human Services
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, Ucl Business Ltd, The Government Of The United States Of America As Represented By The Secretary, Department Of Health And Human Services filed Critical Massachusetts Eye & Ear Infirmary
Publication of PL3821912T3 publication Critical patent/PL3821912T3/pl

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)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Toxicology (AREA)
  • Biotechnology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (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)
  • Saccharide Compounds (AREA)
  • Compounds Of Unknown Constitution (AREA)
PL20199208.8T 2014-07-24 2015-07-17 Terapia genowa rpgr w leczeniu retinopatii barwnikowej PL3821912T3 (pl)

Applications Claiming Priority (1)

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

Publications (1)

Publication Number Publication Date
PL3821912T3 true PL3821912T3 (pl) 2024-10-28

Family

ID=55163577

Family Applications (2)

Application Number Title Priority Date Filing Date
PL20199208.8T PL3821912T3 (pl) 2014-07-24 2015-07-17 Terapia genowa rpgr w leczeniu retinopatii barwnikowej
PL15825383T PL3191139T3 (pl) 2014-07-24 2015-07-17 Terapia genowa RPGR w leczeniu retinopatii barwnikowej

Family Applications After (1)

Application Number Title Priority Date Filing Date
PL15825383T PL3191139T3 (pl) 2014-07-24 2015-07-17 Terapia genowa RPGR w leczeniu retinopatii barwnikowej

Country Status (18)

Country Link
US (2) US10314924B2 (cg-RX-API-DMAC7.html)
EP (2) EP3191139B1 (cg-RX-API-DMAC7.html)
JP (3) JP6654760B2 (cg-RX-API-DMAC7.html)
CN (1) CN107206105A (cg-RX-API-DMAC7.html)
CA (1) CA2991750C (cg-RX-API-DMAC7.html)
CY (1) CY1123793T1 (cg-RX-API-DMAC7.html)
DK (2) DK3191139T3 (cg-RX-API-DMAC7.html)
ES (2) ES2987090T3 (cg-RX-API-DMAC7.html)
FI (1) FI3821912T3 (cg-RX-API-DMAC7.html)
HR (2) HRP20241154T1 (cg-RX-API-DMAC7.html)
HU (2) HUE052781T2 (cg-RX-API-DMAC7.html)
LT (2) LT3191139T (cg-RX-API-DMAC7.html)
PL (2) PL3821912T3 (cg-RX-API-DMAC7.html)
PT (2) PT3821912T (cg-RX-API-DMAC7.html)
RS (2) RS61307B1 (cg-RX-API-DMAC7.html)
SI (2) SI3191139T1 (cg-RX-API-DMAC7.html)
SM (2) SMT202400309T1 (cg-RX-API-DMAC7.html)
WO (1) WO2016014353A1 (cg-RX-API-DMAC7.html)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE052781T2 (hu) * 2014-07-24 2021-05-28 Massachusetts Eye & Ear Infirmary RPGR génterápia a retinitis pigmentosa terápiájára
WO2016098078A2 (en) * 2014-12-19 2016-06-23 Novartis Ag Dimerization switches and uses thereof
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
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
KR102763312B1 (ko) * 2017-10-20 2025-02-07 인쎄름 (엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔) 치료적으로 유효한 양의 재조합 aav9- 유도 벡터의 망막하 전달을 포함하는 대상체의 원추 광수용체 내 관심의 폴리뉴클레오티드를 발현하는 방법
DK3710590T3 (da) * 2017-11-15 2024-08-12 Univ Michigan Regents Virale vektorer omfattende rdh12-kodende områder og fremgangsmåder til behandling af nethindedystrofier
WO2021183779A1 (en) 2020-03-11 2021-09-16 Massachusetts Eye And Ear Infirmary Gene therapy for nmnat1-associated retinal degeneration
CN120350069A (zh) * 2024-01-12 2025-07-22 北京中因科技有限公司 视网膜色素变性动物模型及其构建方法和应用

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
ATE68013T1 (de) 1985-07-05 1991-10-15 Whitehead Biomedical Inst Expression von fremdem genetischem material in epithelzellen.
WO1989002468A1 (en) 1987-09-11 1989-03-23 Whitehead Institute For Biomedical Research Transduced fibroblasts and uses therefor
WO1989005345A1 (en) 1987-12-11 1989-06-15 Whitehead Institute For Biomedical Research Genetic modification of endothelial cells
ATE152169T1 (de) 1988-02-05 1997-05-15 Whitehead Biomedical 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
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
GB201412011D0 (en) * 2014-07-04 2014-08-20 Ucl Business Plc Treatments
HUE052781T2 (hu) * 2014-07-24 2021-05-28 Massachusetts Eye & Ear Infirmary RPGR génterápia a retinitis pigmentosa terápiájára

Also Published As

Publication number Publication date
EP3191139B1 (en) 2020-10-07
ES2987090T3 (es) 2024-11-13
EP3821912A1 (en) 2021-05-19
JP2017523239A (ja) 2017-08-17
US11045558B2 (en) 2021-06-29
CY1123793T1 (el) 2022-05-27
EP3191139A4 (en) 2018-01-10
CN107206105A (zh) 2017-09-26
US10314924B2 (en) 2019-06-11
WO2016014353A1 (en) 2016-01-28
US20200215203A1 (en) 2020-07-09
RS61307B1 (sr) 2021-02-26
ES2834402T3 (es) 2021-06-17
SMT202400309T1 (it) 2024-11-15
CA2991750C (en) 2023-02-14
JP7198329B2 (ja) 2022-12-28
JP2022009333A (ja) 2022-01-14
JP2020073536A (ja) 2020-05-14
JP6654760B2 (ja) 2020-02-26
LT3191139T (lt) 2021-01-25
LT3821912T (lt) 2024-09-25
RS65909B1 (sr) 2024-10-31
HUE052781T2 (hu) 2021-05-28
FI3821912T3 (fi) 2024-08-05
HUE068183T2 (hu) 2024-12-28
PL3191139T3 (pl) 2021-07-05
PT3821912T (pt) 2024-08-01
EP3821912B1 (en) 2024-06-12
SMT202000715T1 (it) 2021-01-05
CA2991750A1 (en) 2016-01-28
SI3821912T1 (sl) 2024-10-30
PT3191139T (pt) 2020-11-05
US20170216454A1 (en) 2017-08-03
DK3821912T3 (da) 2024-08-05
EP3191139A1 (en) 2017-07-19
HRP20241154T1 (hr) 2024-11-22
SI3191139T1 (sl) 2021-03-31
HRP20202023T1 (hr) 2021-03-05
JP6966532B2 (ja) 2021-11-17
DK3191139T3 (da) 2020-11-16

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