HRP20202023T1 - Genska terapija rpgr za liječenje retinitis pigmentose - Google Patents

Genska terapija rpgr za liječenje retinitis pigmentose Download PDF

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Publication number
HRP20202023T1
HRP20202023T1 HRP20202023TT HRP20202023T HRP20202023T1 HR P20202023 T1 HRP20202023 T1 HR P20202023T1 HR P20202023T T HRP20202023T T HR P20202023TT HR P20202023 T HRP20202023 T HR P20202023T HR P20202023 T1 HRP20202023 T1 HR P20202023T1
Authority
HR
Croatia
Prior art keywords
viral vector
rpgr
seq
nucleic acid
cdna
Prior art date
Application number
HRP20202023TT
Other languages
English (en)
Croatian (hr)
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 The Office of Technology Transfer National Institutes 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 and Human Services The Office of Technology Transfer National Institutes of Health filed Critical Massachusetts Eye & Ear Infirmary
Publication of HRP20202023T1 publication Critical patent/HRP20202023T1/hr

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)
HRP20202023TT 2014-07-24 2015-07-17 Genska terapija rpgr za liječenje retinitis pigmentose HRP20202023T1 (hr)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
HRP20202023T1 true HRP20202023T1 (hr) 2021-03-05

Family

ID=55163577

Family Applications (2)

Application Number Title Priority Date Filing Date
HRP20202023TT HRP20202023T1 (hr) 2014-07-24 2015-07-17 Genska terapija rpgr za liječenje retinitis pigmentose
HRP20241154TT HRP20241154T1 (hr) 2014-07-24 2015-07-17 Rpgr genska terapija za pigmentnu retinopatiju

Family Applications After (1)

Application Number Title Priority Date Filing Date
HRP20241154TT HRP20241154T1 (hr) 2014-07-24 2015-07-17 Rpgr genska terapija za pigmentnu retinopatiju

Country Status (18)

Country Link
US (2) US10314924B2 (cg-RX-API-DMAC7.html)
EP (2) EP3821912B1 (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) DK3821912T3 (cg-RX-API-DMAC7.html)
ES (2) ES2834402T3 (cg-RX-API-DMAC7.html)
FI (1) FI3821912T3 (cg-RX-API-DMAC7.html)
HR (2) HRP20202023T1 (cg-RX-API-DMAC7.html)
HU (2) HUE052781T2 (cg-RX-API-DMAC7.html)
LT (2) LT3821912T (cg-RX-API-DMAC7.html)
PL (2) PL3821912T3 (cg-RX-API-DMAC7.html)
PT (2) PT3191139T (cg-RX-API-DMAC7.html)
RS (2) RS65909B1 (cg-RX-API-DMAC7.html)
SI (2) SI3821912T1 (cg-RX-API-DMAC7.html)
SM (2) SMT202000715T1 (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
JP6654760B2 (ja) * 2014-07-24 2020-02-26 マサチューセッツ・アイ・アンド・イア・インファーマリー 網膜色素変性症のためのｒｐｇｒ遺伝子治療
WO2016098078A2 (en) * 2014-12-19 2016-06-23 Novartis Ag Dimerization switches and uses thereof
EP3719134B1 (en) 2015-03-11 2023-08-23 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
RS65881B1 (sr) * 2017-11-15 2024-09-30 Univ Michigan Regents Virusni vektori koji sadrže kodirajuće regione rdh12 i postupci za lečenje distrofija mrežnjače
IL296262A (en) * 2020-03-11 2022-11-01 Massachusetts Eye & Ear Infirmary Gene therapy for nmnat1-related 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
ATE68013T1 (de) 1985-07-05 1991-10-15 Whitehead Biomedical Inst Expression von fremdem genetischem material in epithelzellen.
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
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
CA2095256A1 (en) 1990-10-31 1992-05-01 Brad Guild 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
CN105120901A (zh) 2012-07-11 2015-12-02 宾夕法尼亚大学托管会 Rpgr x染色体连锁视网膜退化的aav介导的基因治疗
GB201412011D0 (en) * 2014-07-04 2014-08-20 Ucl Business Plc Treatments
JP6654760B2 (ja) * 2014-07-24 2020-02-26 マサチューセッツ・アイ・アンド・イア・インファーマリー 網膜色素変性症のためのｒｐｇｒ遺伝子治療

Also Published As

Publication number Publication date
EP3821912B1 (en) 2024-06-12
JP2022009333A (ja) 2022-01-14
RS61307B1 (sr) 2021-02-26
JP6654760B2 (ja) 2020-02-26
CN107206105A (zh) 2017-09-26
CY1123793T1 (el) 2022-05-27
EP3191139A4 (en) 2018-01-10
SMT202000715T1 (it) 2021-01-05
SMT202400309T1 (it) 2024-11-15
FI3821912T3 (fi) 2024-08-05
EP3191139A1 (en) 2017-07-19
LT3821912T (lt) 2024-09-25
DK3191139T3 (da) 2020-11-16
CA2991750A1 (en) 2016-01-28
CA2991750C (en) 2023-02-14
HUE068183T2 (hu) 2024-12-28
JP2020073536A (ja) 2020-05-14
LT3191139T (lt) 2021-01-25
US20170216454A1 (en) 2017-08-03
JP2017523239A (ja) 2017-08-17
WO2016014353A1 (en) 2016-01-28
ES2834402T3 (es) 2021-06-17
US20200215203A1 (en) 2020-07-09
SI3821912T1 (sl) 2024-10-30
EP3821912A1 (en) 2021-05-19
JP7198329B2 (ja) 2022-12-28
DK3821912T3 (da) 2024-08-05
US10314924B2 (en) 2019-06-11
RS65909B1 (sr) 2024-10-31
US11045558B2 (en) 2021-06-29
PL3191139T3 (pl) 2021-07-05
PT3821912T (pt) 2024-08-01
EP3191139B1 (en) 2020-10-07
HUE052781T2 (hu) 2021-05-28
SI3191139T1 (sl) 2021-03-31
PL3821912T3 (pl) 2024-10-28
PT3191139T (pt) 2020-11-05
JP6966532B2 (ja) 2021-11-17
ES2987090T3 (es) 2024-11-13
HRP20241154T1 (hr) 2024-11-22

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