RS61307B1 - Rpgr genska terapija za pigmentnu retinopatiju - Google Patents

Rpgr genska terapija za pigmentnu retinopatiju

Info

Publication number
RS61307B1
RS61307B1 RS20201416A RSP20201416A RS61307B1 RS 61307 B1 RS61307 B1 RS 61307B1 RS 20201416 A RS20201416 A RS 20201416A RS P20201416 A RSP20201416 A RS P20201416A RS 61307 B1 RS61307 B1 RS 61307B1
Authority
RS
Serbia
Prior art keywords
gene therapy
retinitis pigmentosa
rpgr gene
rpgr
pigmentosa
Prior art date
Application number
RS20201416A
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
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 RS61307B1 publication Critical patent/RS61307B1/sr

Links

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)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (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)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Saccharide Compounds (AREA)
  • Compounds Of Unknown Constitution (AREA)
RS20201416A 2014-07-24 2015-07-17 Rpgr genska terapija za pigmentnu retinopatiju RS61307B1 (sr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462028638P 2014-07-24 2014-07-24
EP15825383.1A EP3191139B1 (en) 2014-07-24 2015-07-17 Rpgr gene therapy for retinitis pigmentosa
PCT/US2015/040866 WO2016014353A1 (en) 2014-07-24 2015-07-17 Rpgr gene therapy for retinitis pigmentosa

Publications (1)

Publication Number Publication Date
RS61307B1 true RS61307B1 (sr) 2021-02-26

Family

ID=55163577

Family Applications (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 (16)

Country Link
US (2) US10314924B2 (sr)
EP (1) EP3191139B1 (sr)
JP (3) JP6654760B2 (sr)
CN (1) CN107206105A (sr)
CA (1) CA2991750C (sr)
CY (1) CY1123793T1 (sr)
DK (1) DK3191139T3 (sr)
ES (1) ES2834402T3 (sr)
HR (1) HRP20202023T1 (sr)
HU (1) HUE052781T2 (sr)
LT (1) LT3191139T (sr)
PL (1) PL3191139T3 (sr)
PT (1) PT3191139T (sr)
RS (1) RS61307B1 (sr)
SI (1) SI3191139T1 (sr)
WO (1) WO2016014353A1 (sr)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2834402T3 (es) 2014-07-24 2021-06-17 Massachusetts Eye & Ear Infirmary Terapia génica con RPGR para retinitis pigmentosa
US20190054117A1 (en) * 2014-12-19 2019-02-21 Novartis Ag Dimerization switches and uses thereof
US10646588B2 (en) 2015-03-11 2020-05-12 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services RP2 and 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
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
WO2019099696A1 (en) * 2017-11-15 2019-05-23 The Regents Of The University Of Michigan Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies
CN115379834A (zh) * 2020-03-11 2022-11-22 马萨诸塞眼科耳科诊所 用于nmnat1相关视网膜变性的基因疗法

Family Cites Families (14)

* 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.
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
DE3852823T2 (de) 1987-09-11 1995-05-24 Whitehead Biomedical Inst Transduktionsveränderte fibroblasten und ihre anwendung.
JP2914692B2 (ja) 1987-12-11 1999-07-05 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 内皮細胞の遺伝子修飾
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
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
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
AU2013287281B2 (en) * 2012-07-11 2018-04-26 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
ES2834402T3 (es) * 2014-07-24 2021-06-17 Massachusetts Eye & Ear Infirmary Terapia génica con RPGR para retinitis pigmentosa

Also Published As

Publication number Publication date
HRP20202023T1 (hr) 2021-03-05
PT3191139T (pt) 2020-11-05
EP3191139B1 (en) 2020-10-07
EP3191139A1 (en) 2017-07-19
US20200215203A1 (en) 2020-07-09
JP7198329B2 (ja) 2022-12-28
CY1123793T1 (el) 2022-05-27
EP3821912A1 (en) 2021-05-19
SI3191139T1 (sl) 2021-03-31
JP2020073536A (ja) 2020-05-14
CA2991750C (en) 2023-02-14
CA2991750A1 (en) 2016-01-28
US10314924B2 (en) 2019-06-11
JP2022009333A (ja) 2022-01-14
US11045558B2 (en) 2021-06-29
PL3191139T3 (pl) 2021-07-05
DK3191139T3 (da) 2020-11-16
HUE052781T2 (hu) 2021-05-28
WO2016014353A1 (en) 2016-01-28
EP3191139A4 (en) 2018-01-10
US20170216454A1 (en) 2017-08-03
CN107206105A (zh) 2017-09-26
JP6654760B2 (ja) 2020-02-26
JP6966532B2 (ja) 2021-11-17
JP2017523239A (ja) 2017-08-17
ES2834402T3 (es) 2021-06-17
LT3191139T (lt) 2021-01-25

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