CY1123793T1 - Γονιδιακη θεραπεια rpgr για μελαγχρωστικη αμφιβληστροειδοπαθεια - Google Patents

Γονιδιακη θεραπεια rpgr για μελαγχρωστικη αμφιβληστροειδοπαθεια

Info

Publication number
CY1123793T1
CY1123793T1 CY20211100002T CY211100002T CY1123793T1 CY 1123793 T1 CY1123793 T1 CY 1123793T1 CY 20211100002 T CY20211100002 T CY 20211100002T CY 211100002 T CY211100002 T CY 211100002T CY 1123793 T1 CY1123793 T1 CY 1123793T1
Authority
CY
Cyprus
Prior art keywords
rpgr
melangrosis
amphiblistroidopathy
gene therapy
rpgr gene
Prior art date
Application number
CY20211100002T
Other languages
Greek (el)
English (en)
Inventor
Michael A. Sandberg
Basil PAWLYK
Tiansen Li
Xinhua SHU
Alan Finlay WRIGHT
Robin Ali
Original Assignee
Massachusetts Eye & Ear Infirmary
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 filed Critical Massachusetts Eye & Ear Infirmary
Publication of CY1123793T1 publication Critical patent/CY1123793T1/el

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)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Saccharide Compounds (AREA)
  • Compounds Of Unknown Constitution (AREA)
CY20211100002T 2014-07-24 2021-01-05 Γονιδιακη θεραπεια rpgr για μελαγχρωστικη αμφιβληστροειδοπαθεια CY1123793T1 (el)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
CY1123793T1 true CY1123793T1 (el) 2022-05-27

Family

ID=55163577

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20211100002T CY1123793T1 (el) 2014-07-24 2021-01-05 Γονιδιακη θεραπεια rpgr για μελαγχρωστικη αμφιβληστροειδοπαθεια

Country Status (18)

Country Link
US (2) US10314924B2 (2)
EP (2) EP3191139B1 (2)
JP (3) JP6654760B2 (2)
CN (1) CN107206105A (2)
CA (1) CA2991750C (2)
CY (1) CY1123793T1 (2)
DK (2) DK3191139T3 (2)
ES (2) ES2987090T3 (2)
FI (1) FI3821912T3 (2)
HR (2) HRP20241154T1 (2)
HU (2) HUE052781T2 (2)
LT (2) LT3821912T (2)
PL (2) PL3821912T3 (2)
PT (2) PT3191139T (2)
RS (2) RS61307B1 (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
PT3191139T (pt) * 2014-07-24 2020-11-05 Massachusetts Eye & Ear Infirmary Terapia do gene rpgr para retinite pigmentosa
US20190054117A1 (en) * 2014-12-19 2019-02-21 Novartis Ag Dimerization switches and uses thereof
AU2016228751B2 (en) * 2015-03-11 2022-01-13 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
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
SG11202004527YA (en) 2017-11-15 2020-06-29 Univ Michigan Regents Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies
US12257320B2 (en) 2020-03-11 2025-03-25 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
WO1987000201A1 (en) 1985-07-05 1987-01-15 Whitehead Institute For Biomedical Research Epithelial cells expressing foreign genetic material
EP0378576B1 (en) 1987-09-11 1995-01-18 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
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
JP3249516B2 (ja) 1990-10-31 2002-01-21 ソマティクス セラピー コーポレイション 遺伝子治療のためのレトロウイルスのベクター
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
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
PT3191139T (pt) * 2014-07-24 2020-11-05 Massachusetts Eye & Ear Infirmary Terapia do gene rpgr para retinite pigmentosa

Also Published As

Publication number Publication date
SI3191139T1 (sl) 2021-03-31
JP7198329B2 (ja) 2022-12-28
FI3821912T3 (fi) 2024-08-05
CA2991750C (en) 2023-02-14
EP3191139A1 (en) 2017-07-19
JP2020073536A (ja) 2020-05-14
CA2991750A1 (en) 2016-01-28
SI3821912T1 (sl) 2024-10-30
SMT202400309T1 (it) 2024-11-15
US20170216454A1 (en) 2017-08-03
JP2022009333A (ja) 2022-01-14
EP3191139B1 (en) 2020-10-07
HUE052781T2 (hu) 2021-05-28
EP3821912A1 (en) 2021-05-19
PL3191139T3 (pl) 2021-07-05
PT3821912T (pt) 2024-08-01
PL3821912T3 (pl) 2024-10-28
US20200215203A1 (en) 2020-07-09
DK3191139T3 (da) 2020-11-16
ES2987090T3 (es) 2024-11-13
US10314924B2 (en) 2019-06-11
LT3191139T (lt) 2021-01-25
HRP20241154T1 (hr) 2024-11-22
EP3191139A4 (en) 2018-01-10
PT3191139T (pt) 2020-11-05
US11045558B2 (en) 2021-06-29
DK3821912T3 (da) 2024-08-05
RS65909B1 (sr) 2024-10-31
EP3821912B1 (en) 2024-06-12
JP6654760B2 (ja) 2020-02-26
RS61307B1 (sr) 2021-02-26
SMT202000715T1 (it) 2021-01-05
JP6966532B2 (ja) 2021-11-17
WO2016014353A1 (en) 2016-01-28
LT3821912T (lt) 2024-09-25
CN107206105A (zh) 2017-09-26
JP2017523239A (ja) 2017-08-17
HRP20202023T1 (hr) 2021-03-05
HUE068183T2 (hu) 2024-12-28
ES2834402T3 (es) 2021-06-17

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