PT3191139T - Terapia do gene rpgr para retinite pigmentosa - Google Patents
Terapia do gene rpgr para retinite pigmentosaInfo
- Publication number
- PT3191139T PT3191139T PT158253831T PT15825383T PT3191139T PT 3191139 T PT3191139 T PT 3191139T PT 158253831 T PT158253831 T PT 158253831T PT 15825383 T PT15825383 T PT 15825383T PT 3191139 T PT3191139 T PT 3191139T
- Authority
- PT
- Portugal
- Prior art keywords
- gene therapy
- retinitis pigmentosa
- rpgr gene
- rpgr
- pigmentosa
- Prior art date
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)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Engineering & Computer Science (AREA)
- Gastroenterology & Hepatology (AREA)
- Zoology (AREA)
- Toxicology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biotechnology (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)
- Medicinal Preparation (AREA)
- Peptides Or Proteins (AREA)
- Saccharide Compounds (AREA)
- Compounds Of Unknown Constitution (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462028638P | 2014-07-24 | 2014-07-24 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PT3191139T true PT3191139T (pt) | 2020-11-05 |
Family
ID=55163577
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT201992088T PT3821912T (pt) | 2014-07-24 | 2015-07-17 | Terapia do gene rpgr para retinite pigmentosa |
| PT158253831T PT3191139T (pt) | 2014-07-24 | 2015-07-17 | Terapia do gene rpgr para retinite pigmentosa |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT201992088T PT3821912T (pt) | 2014-07-24 | 2015-07-17 | Terapia do gene rpgr para retinite pigmentosa |
Country Status (17)
| Country | Link |
|---|---|
| US (2) | US10314924B2 (pt) |
| EP (2) | EP3821912B1 (pt) |
| JP (3) | JP6654760B2 (pt) |
| CN (1) | CN107206105A (pt) |
| CA (1) | CA2991750C (pt) |
| CY (1) | CY1123793T1 (pt) |
| DK (2) | DK3821912T3 (pt) |
| ES (1) | ES2834402T3 (pt) |
| FI (1) | FI3821912T3 (pt) |
| HR (1) | HRP20202023T1 (pt) |
| HU (1) | HUE052781T2 (pt) |
| LT (2) | LT3191139T (pt) |
| PL (1) | PL3191139T3 (pt) |
| PT (2) | PT3821912T (pt) |
| RS (1) | RS61307B1 (pt) |
| SI (1) | SI3191139T1 (pt) |
| WO (1) | WO2016014353A1 (pt) |
Families Citing this family (8)
| 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 |
| US20190054117A1 (en) * | 2014-12-19 | 2019-02-21 | Novartis Ag | Dimerization switches and uses thereof |
| WO2016145345A1 (en) * | 2015-03-11 | 2016-09-15 | 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 |
| 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 |
| EP4364803A3 (en) * | 2017-11-15 | 2024-06-05 | The Regents of the University of Michigan | Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies |
| AU2021236233A1 (en) * | 2020-03-11 | 2022-09-22 | Massachusetts Eye And Ear Infirmary | Gene therapy for NMNAT1-associated retinal degeneration |
Family Cites Families (14)
| 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 |
| EP0228458B2 (en) | 1985-07-05 | 1997-10-22 | Whitehead Institute For Biomedical Research | Epithelial cells expressing foreign genetic material |
| 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 | Hughes Howard Med Inst | Transduktionsveränderte fibroblasten und ihre anwendung. |
| WO1989005345A1 (en) | 1987-12-11 | 1989-06-15 | Whitehead Institute For Biomedical Research | Genetic modification of endothelial cells |
| DE68927996T2 (de) | 1988-02-05 | 1997-12-04 | Hughes Howard Med Inst | Modifizierte hepatozyten und deren anwendung |
| DK0556345T3 (da) | 1990-10-31 | 1997-06-16 | Whitehead Biomedical Inst | Retrovirale vektorer,som er egnede til genterapi |
| 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 |
| 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 |
| 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 |
-
2015
- 2015-07-17 JP JP2017525514A patent/JP6654760B2/ja active Active
- 2015-07-17 DK DK20199208.8T patent/DK3821912T3/da active
- 2015-07-17 LT LTEP15825383.1T patent/LT3191139T/lt unknown
- 2015-07-17 EP EP20199208.8A patent/EP3821912B1/en active Active
- 2015-07-17 US US15/328,617 patent/US10314924B2/en active Active
- 2015-07-17 ES ES15825383T patent/ES2834402T3/es active Active
- 2015-07-17 HU HUE15825383A patent/HUE052781T2/hu unknown
- 2015-07-17 PT PT201992088T patent/PT3821912T/pt unknown
- 2015-07-17 DK DK15825383.1T patent/DK3191139T3/da active
- 2015-07-17 CA CA2991750A patent/CA2991750C/en active Active
- 2015-07-17 EP EP15825383.1A patent/EP3191139B1/en active Active
- 2015-07-17 LT LTEP20199208.8T patent/LT3821912T/lt unknown
- 2015-07-17 PT PT158253831T patent/PT3191139T/pt unknown
- 2015-07-17 PL PL15825383T patent/PL3191139T3/pl unknown
- 2015-07-17 SI SI201531413T patent/SI3191139T1/sl unknown
- 2015-07-17 WO PCT/US2015/040866 patent/WO2016014353A1/en active Application Filing
- 2015-07-17 CN CN201580051512.3A patent/CN107206105A/zh active Pending
- 2015-07-17 FI FIEP20199208.8T patent/FI3821912T3/fi active
- 2015-07-17 RS RS20201416A patent/RS61307B1/sr unknown
-
2019
- 2019-06-10 US US16/436,521 patent/US11045558B2/en active Active
- 2019-12-26 JP JP2019235539A patent/JP6966532B2/ja active Active
-
2020
- 2020-12-17 HR HRP20202023TT patent/HRP20202023T1/hr unknown
-
2021
- 2021-01-05 CY CY20211100002T patent/CY1123793T1/el unknown
- 2021-10-21 JP JP2021172128A patent/JP7198329B2/ja active Active
Also Published As
| Publication number | Publication date |
|---|---|
| US20200215203A1 (en) | 2020-07-09 |
| US20170216454A1 (en) | 2017-08-03 |
| CY1123793T1 (el) | 2022-05-27 |
| EP3191139A1 (en) | 2017-07-19 |
| FI3821912T3 (fi) | 2024-08-05 |
| PL3191139T3 (pl) | 2021-07-05 |
| PT3821912T (pt) | 2024-08-01 |
| US11045558B2 (en) | 2021-06-29 |
| RS61307B1 (sr) | 2021-02-26 |
| HUE052781T2 (hu) | 2021-05-28 |
| LT3821912T (lt) | 2024-09-25 |
| JP7198329B2 (ja) | 2022-12-28 |
| JP2022009333A (ja) | 2022-01-14 |
| JP2017523239A (ja) | 2017-08-17 |
| US10314924B2 (en) | 2019-06-11 |
| EP3821912B1 (en) | 2024-06-12 |
| WO2016014353A1 (en) | 2016-01-28 |
| LT3191139T (lt) | 2021-01-25 |
| CA2991750A1 (en) | 2016-01-28 |
| JP6966532B2 (ja) | 2021-11-17 |
| EP3191139A4 (en) | 2018-01-10 |
| JP2020073536A (ja) | 2020-05-14 |
| DK3821912T3 (da) | 2024-08-05 |
| DK3191139T3 (da) | 2020-11-16 |
| EP3191139B1 (en) | 2020-10-07 |
| HRP20202023T1 (hr) | 2021-03-05 |
| CN107206105A (zh) | 2017-09-26 |
| EP3821912A1 (en) | 2021-05-19 |
| CA2991750C (en) | 2023-02-14 |
| SI3191139T1 (sl) | 2021-03-31 |
| ES2834402T3 (es) | 2021-06-17 |
| JP6654760B2 (ja) | 2020-02-26 |
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