CA2991750C - Rpgr gene therapy for retinitis pigmentosa - Google Patents

Rpgr gene therapy for retinitis pigmentosa Download PDF

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Publication number
CA2991750C
CA2991750C CA2991750A CA2991750A CA2991750C CA 2991750 C CA2991750 C CA 2991750C CA 2991750 A CA2991750 A CA 2991750A CA 2991750 A CA2991750 A CA 2991750A CA 2991750 C CA2991750 C CA 2991750C
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CA
Canada
Prior art keywords
rpgr
nucleic acid
protein
human
acid molecule
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
CA2991750A
Other languages
English (en)
French (fr)
Other versions
CA2991750A1 (en
Inventor
Michael A. Sandberg
Basil PAWLYK
Alan Finlay WRIGHT
Xinhua SHU
Tiansen Li
Robin Ali
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
UCL Business Ltd
Massachusetts Eye and Ear Infirmary
United States Department of Health and Human Services
Original Assignee
UCL Business Ltd
Massachusetts Eye and Ear Infirmary
United States 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 UCL Business Ltd, Massachusetts Eye and Ear Infirmary, United States Department of Health and Human Services filed Critical UCL Business Ltd
Publication of CA2991750A1 publication Critical patent/CA2991750A1/en
Application granted granted Critical
Publication of CA2991750C publication Critical patent/CA2991750C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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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)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Toxicology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Compounds Of Unknown Constitution (AREA)
  • Saccharide Compounds (AREA)
CA2991750A 2014-07-24 2015-07-17 Rpgr gene therapy for retinitis pigmentosa Active CA2991750C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462028638P 2014-07-24 2014-07-24
US62/028,638 2014-07-24
PCT/US2015/040866 WO2016014353A1 (en) 2014-07-24 2015-07-17 Rpgr gene therapy for retinitis pigmentosa

Publications (2)

Publication Number Publication Date
CA2991750A1 CA2991750A1 (en) 2016-01-28
CA2991750C true CA2991750C (en) 2023-02-14

Family

ID=55163577

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2991750A Active CA2991750C (en) 2014-07-24 2015-07-17 Rpgr gene therapy for retinitis pigmentosa

Country Status (18)

Country Link
US (2) US10314924B2 (enExample)
EP (2) EP3821912B1 (enExample)
JP (3) JP6654760B2 (enExample)
CN (2) CN107206105A (enExample)
CA (1) CA2991750C (enExample)
CY (1) CY1123793T1 (enExample)
DK (2) DK3821912T3 (enExample)
ES (2) ES2987090T3 (enExample)
FI (1) FI3821912T3 (enExample)
HR (2) HRP20202023T1 (enExample)
HU (2) HUE052781T2 (enExample)
LT (2) LT3821912T (enExample)
PL (2) PL3191139T3 (enExample)
PT (2) PT3191139T (enExample)
RS (2) RS65909B1 (enExample)
SI (2) SI3821912T1 (enExample)
SM (2) SMT202000715T1 (enExample)
WO (1) WO2016014353A1 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SMT202000715T1 (it) * 2014-07-24 2021-01-05 Massachusetts Eye & Ear Infirmary Terapia genica rpgr per la retinite pigmentosa
WO2016098078A2 (en) * 2014-12-19 2016-06-23 Novartis Ag Dimerization switches and uses thereof
CA2979229A1 (en) 2015-03-11 2016-09-15 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
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
SMT202400321T1 (it) * 2017-11-15 2024-11-15 Univ Michigan Regents Vettori virali comprendenti regioni codificanti rdh12 e metodi di trattamento di distrofie retiniche
MX2022011177A (es) 2020-03-11 2022-12-13 Massachusetts Eye & Ear Infirmary Terapia genica para la degeneracion de la retina asociada a nmnat1.
CA3225080A1 (en) * 2021-07-14 2023-01-19 Anastasios GEORGIADIS Kcnv2 gene therapy
CN117165596A (zh) * 2022-05-27 2023-12-05 武汉纽福斯生物科技有限公司 编码rpgr的核酸及其应用
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
WO2026150224A1 (en) 2024-12-30 2026-07-16 MeiraGTx Ocular UK Limited Modified adeno-associated virus (aav) viral capsid proteins and methods of use

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
DE3681787D1 (de) 1985-07-05 1991-11-07 Whitehead Biomedical Inst Expression von fremdem genetischem material in epithelzellen.
HK1008049A1 (en) 1987-09-11 1999-04-30 Whitehead Institute For Biomedical Research Transduced fibroblasts and use therefor
ATE110108T1 (de) 1987-12-11 1994-09-15 Whitehead Biomedical Inst Genetische modifizierung von endothelialen zellen.
DE68927996T2 (de) 1988-02-05 1997-12-04 Hughes Howard Med Inst Modifizierte hepatozyten und deren anwendung
DE69123981T3 (de) 1990-10-31 2006-06-14 Cell Genesys Inc Nützliche retrovirale vektoren für die gentherapie
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
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
SMT202000715T1 (it) * 2014-07-24 2021-01-05 Massachusetts Eye & Ear Infirmary Terapia genica rpgr per la retinite pigmentosa

Also Published As

Publication number Publication date
JP6966532B2 (ja) 2021-11-17
FI3821912T3 (fi) 2024-08-05
EP3821912B1 (en) 2024-06-12
CY1123793T1 (el) 2022-05-27
ES2987090T3 (es) 2024-11-13
LT3821912T (lt) 2024-09-25
JP2020073536A (ja) 2020-05-14
SMT202400309T1 (it) 2024-11-15
RS61307B1 (sr) 2021-02-26
US20170216454A1 (en) 2017-08-03
EP3821912A1 (en) 2021-05-19
HRP20241154T1 (hr) 2024-11-22
CA2991750A1 (en) 2016-01-28
SI3191139T1 (sl) 2021-03-31
JP7198329B2 (ja) 2022-12-28
US10314924B2 (en) 2019-06-11
JP2017523239A (ja) 2017-08-17
PT3191139T (pt) 2020-11-05
CN107206105A (zh) 2017-09-26
US11045558B2 (en) 2021-06-29
SI3821912T1 (sl) 2024-10-30
SMT202000715T1 (it) 2021-01-05
JP2022009333A (ja) 2022-01-14
PL3191139T3 (pl) 2021-07-05
PT3821912T (pt) 2024-08-01
HRP20202023T1 (hr) 2021-03-05
CN122235151A (zh) 2026-06-19
DK3821912T3 (da) 2024-08-05
PL3821912T3 (pl) 2024-10-28
HUE068183T2 (hu) 2024-12-28
LT3191139T (lt) 2021-01-25
ES2834402T3 (es) 2021-06-17
US20200215203A1 (en) 2020-07-09
EP3191139A1 (en) 2017-07-19
JP6654760B2 (ja) 2020-02-26
EP3191139A4 (en) 2018-01-10
WO2016014353A1 (en) 2016-01-28
DK3191139T3 (da) 2020-11-16
HUE052781T2 (hu) 2021-05-28
RS65909B1 (sr) 2024-10-31
EP3191139B1 (en) 2020-10-07

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