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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Ophthalmology & Optometry (AREA)
  • Biochemistry (AREA)
  • Toxicology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (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)
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 (2)
EP (2) EP3821912B1 (2)
JP (3) JP6654760B2 (2)
CN (1) CN107206105A (2)
CA (1) CA2991750C (2)
CY (1) CY1123793T1 (2)
DK (2) DK3821912T3 (2)
ES (2) ES2987090T3 (2)
FI (1) FI3821912T3 (2)
HR (2) HRP20202023T1 (2)
HU (2) HUE068183T2 (2)
LT (2) LT3821912T (2)
PL (2) PL3821912T3 (2)
PT (2) PT3191139T (2)
RS (2) RS65909B1 (2)
SI (2) SI3191139T1 (2)
SM (2) SMT202000715T1 (2)
WO (1) WO2016014353A1 (2)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE068183T2 (hu) * 2014-07-24 2024-12-28 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
CN111867635A (zh) * 2017-10-20 2020-10-30 法国国家卫生及研究医学协会 包括视网膜下递送治疗有效量的重组aav9衍生载体的在对象的视锥细胞光感受器中表达目的多核苷酸的方法
KR102704296B1 (ko) * 2017-11-15 2024-09-05 더 리젠츠 오브 더 유니버시티 오브 미시간 Rdh12 암호화 영역을 포함하는 바이러스 벡터 및 망막 이영양증을 치료하는 방법
EP4117644A4 (en) 2020-03-11 2024-08-14 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
IL79289A (en) 1985-07-05 1992-01-15 Whitehead Biomedical Inst Introduction and expression of foreign genetic material into keratinocytes using a recombinant retrovirus
ATE117375T1 (de) 1987-09-11 1995-02-15 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
WO1989007136A2 (en) 1988-02-05 1989-08-10 Whitehead Institute For Biomedical Research Modified hepatocytes and uses therefor
ES2096750T3 (es) 1990-10-31 1997-03-16 Somatix Therapy Corp Vectores retroviricos utiles para la terapia genica.
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
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
HUE068183T2 (hu) * 2014-07-24 2024-12-28 Massachusetts Eye & Ear Infirmary RPGR génterápia a retinitis pigmentosa terápiájára

Also Published As

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

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