RS65909B1 - Rpgr genska terapija za pigmentnu retinopatiju - Google Patents

Rpgr genska terapija za pigmentnu retinopatiju

Info

Publication number
RS65909B1
RS65909B1 RS20240901A RSP20240901A RS65909B1 RS 65909 B1 RS65909 B1 RS 65909B1 RS 20240901 A RS20240901 A RS 20240901A RS P20240901 A RSP20240901 A RS P20240901A RS 65909 B1 RS65909 B1 RS 65909B1
Authority
RS
Serbia
Prior art keywords
glu
gly
rpgr
nucleic acid
viral vector
Prior art date
Application number
RS20240901A
Other languages
English (en)
Serbian (sr)
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
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 filed Critical Massachusetts Eye & Ear Infirmary
Publication of RS65909B1 publication Critical patent/RS65909B1/sr

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)
RS20240901A 2014-07-24 2015-07-17 Rpgr genska terapija za pigmentnu retinopatiju RS65909B1 (sr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462028638P 2014-07-24 2014-07-24
EP20199208.8A EP3821912B1 (en) 2014-07-24 2015-07-17 Rpgr gene therapy for retinitis pigmentosa

Publications (1)

Publication Number Publication Date
RS65909B1 true RS65909B1 (sr) 2024-10-31

Family

ID=55163577

Family Applications (2)

Application Number Title Priority Date Filing Date
RS20240901A RS65909B1 (sr) 2014-07-24 2015-07-17 Rpgr genska terapija za pigmentnu retinopatiju
RS20201416A RS61307B1 (sr) 2014-07-24 2015-07-17 Rpgr genska terapija za pigmentnu retinopatiju

Family Applications After (1)

Application Number Title Priority Date Filing Date
RS20201416A RS61307B1 (sr) 2014-07-24 2015-07-17 Rpgr genska terapija za pigmentnu retinopatiju

Country Status (18)

Country Link
US (2) US10314924B2 (enExample)
EP (2) EP3821912B1 (enExample)
JP (3) JP6654760B2 (enExample)
CN (1) CN107206105A (enExample)
CA (1) CA2991750C (enExample)
CY (1) CY1123793T1 (enExample)
DK (2) DK3821912T3 (enExample)
ES (2) ES2987090T3 (enExample)
FI (1) FI3821912T3 (enExample)
HR (2) HRP20241154T1 (enExample)
HU (2) HUE052781T2 (enExample)
LT (2) LT3821912T (enExample)
PL (2) PL3821912T3 (enExample)
PT (2) PT3191139T (enExample)
RS (2) RS65909B1 (enExample)
SI (2) SI3821912T1 (enExample)
SM (2) SMT202400309T1 (enExample)
WO (1) WO2016014353A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RS65909B1 (sr) * 2014-07-24 2024-10-31 Massachusetts Eye & Ear Infirmary Rpgr genska terapija za pigmentnu retinopatiju
WO2016098078A2 (en) * 2014-12-19 2016-06-23 Novartis Ag Dimerization switches and uses thereof
JP6935049B2 (ja) 2015-03-11 2021-09-15 アメリカ合衆国 Ｘ連鎖性網膜色素変性症の治療のためのｒｐ２及びｒｐｇｒベクター
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
JP7420710B2 (ja) * 2017-10-20 2024-01-23 アンセルム（アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル） 組換えａａｖ９由来ベクターの治療有効量の網膜下送達を含む、対象の錐体視細胞において目的のポリヌクレオチドを発現させる方法
HRP20241073T1 (hr) * 2017-11-15 2024-11-08 The Regents Of The University Of Michigan Virusni vektori koji sadrže kodirajuće regije rdh12 i postupci za liječenje distrofija mrežnice
AU2021236233A1 (en) * 2020-03-11 2022-09-22 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
IL79289A (en) 1985-07-05 1992-01-15 Whitehead Biomedical Inst Introduction and expression of foreign genetic material into keratinocytes using a recombinant retrovirus
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
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
JP2917998B2 (ja) 1988-02-05 1999-07-12 ホワイトヘッド・インスティチュート・フォー・バイオメディカル・リサーチ 修飾された肝細胞およびその用途
WO1992007943A1 (en) 1990-10-31 1992-05-14 Somatix Therapy Corporation Retroviral vectors useful for gene therapy
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
NZ704275A (en) 2012-07-11 2016-09-30 Univ Florida Aav-mediated gene therapy for rpgr x-linked retinal degeneration
GB201412011D0 (en) * 2014-07-04 2014-08-20 Ucl Business Plc Treatments
RS65909B1 (sr) * 2014-07-24 2024-10-31 Massachusetts Eye & Ear Infirmary Rpgr genska terapija za pigmentnu retinopatiju

Also Published As

Publication number Publication date
LT3191139T (lt) 2021-01-25
US20170216454A1 (en) 2017-08-03
ES2834402T3 (es) 2021-06-17
HRP20202023T1 (hr) 2021-03-05
EP3191139A1 (en) 2017-07-19
CA2991750C (en) 2023-02-14
CN107206105A (zh) 2017-09-26
JP2020073536A (ja) 2020-05-14
JP7198329B2 (ja) 2022-12-28
ES2987090T3 (es) 2024-11-13
HUE068183T2 (hu) 2024-12-28
PL3821912T3 (pl) 2024-10-28
US10314924B2 (en) 2019-06-11
JP2017523239A (ja) 2017-08-17
FI3821912T3 (fi) 2024-08-05
EP3191139B1 (en) 2020-10-07
DK3191139T3 (da) 2020-11-16
US11045558B2 (en) 2021-06-29
DK3821912T3 (da) 2024-08-05
US20200215203A1 (en) 2020-07-09
LT3821912T (lt) 2024-09-25
SI3191139T1 (sl) 2021-03-31
JP2022009333A (ja) 2022-01-14
PL3191139T3 (pl) 2021-07-05
PT3821912T (pt) 2024-08-01
JP6654760B2 (ja) 2020-02-26
EP3821912A1 (en) 2021-05-19
JP6966532B2 (ja) 2021-11-17
CY1123793T1 (el) 2022-05-27
SMT202400309T1 (it) 2024-11-15
EP3821912B1 (en) 2024-06-12
SI3821912T1 (sl) 2024-10-30
CA2991750A1 (en) 2016-01-28
EP3191139A4 (en) 2018-01-10
WO2016014353A1 (en) 2016-01-28
RS61307B1 (sr) 2021-02-26
SMT202000715T1 (it) 2021-01-05
HRP20241154T1 (hr) 2024-11-22
HUE052781T2 (hu) 2021-05-28
PT3191139T (pt) 2020-11-05

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