HRP20241154T1 - Rpgr genska terapija za pigmentnu retinopatiju - Google Patents
Rpgr genska terapija za pigmentnu retinopatiju Download PDFInfo
- Publication number
- HRP20241154T1 HRP20241154T1 HRP20241154TT HRP20241154T HRP20241154T1 HR P20241154 T1 HRP20241154 T1 HR P20241154T1 HR P20241154T T HRP20241154T T HR P20241154TT HR P20241154 T HRP20241154 T HR P20241154T HR P20241154 T1 HRP20241154 T1 HR P20241154T1
- Authority
- HR
- Croatia
- Prior art keywords
- viral vector
- nucleic acid
- protein
- truncated human
- rpgr
- Prior art date
Links
- 208000007014 Retinitis pigmentosa Diseases 0.000 title claims 4
- 238000001415 gene therapy Methods 0.000 title 1
- 239000013603 viral vector Substances 0.000 claims 18
- 102000039446 nucleic acids Human genes 0.000 claims 17
- 108020004707 nucleic acids Proteins 0.000 claims 17
- 150000007523 nucleic acids Chemical class 0.000 claims 17
- 101001104102 Homo sapiens X-linked retinitis pigmentosa GTPase regulator Proteins 0.000 claims 13
- 102000050188 human RPGR Human genes 0.000 claims 13
- 108090000623 proteins and genes Proteins 0.000 claims 6
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 4
- 102000013446 GTP Phosphohydrolases Human genes 0.000 claims 3
- 108091006109 GTPases Proteins 0.000 claims 3
- 208000017442 Retinal disease Diseases 0.000 claims 3
- 206010038923 Retinopathy Diseases 0.000 claims 3
- 230000004777 loss-of-function mutation Effects 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- 230000001105 regulatory effect Effects 0.000 claims 3
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 102000004437 G-Protein-Coupled Receptor Kinase 1 Human genes 0.000 claims 2
- 101000611338 Homo sapiens Rhodopsin Proteins 0.000 claims 2
- 206010025421 Macule Diseases 0.000 claims 1
- 239000012530 fluid Substances 0.000 claims 1
- 238000000520 microinjection Methods 0.000 claims 1
- 210000001328 optic nerve Anatomy 0.000 claims 1
- 230000002123 temporal effect Effects 0.000 claims 1
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)
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 |
|---|---|
| HRP20241154T1 true HRP20241154T1 (hr) | 2024-11-22 |
Family
ID=55163577
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20241154TT HRP20241154T1 (hr) | 2014-07-24 | 2015-07-17 | Rpgr genska terapija za pigmentnu retinopatiju |
| HRP20202023TT HRP20202023T1 (hr) | 2014-07-24 | 2015-07-17 | Genska terapija rpgr za liječenje retinitis pigmentose |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20202023TT HRP20202023T1 (hr) | 2014-07-24 | 2015-07-17 | Genska terapija rpgr za liječenje retinitis pigmentose |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US10314924B2 (enExample) |
| EP (2) | EP3191139B1 (enExample) |
| JP (3) | JP6654760B2 (enExample) |
| CN (1) | CN107206105A (enExample) |
| CA (1) | CA2991750C (enExample) |
| CY (1) | CY1123793T1 (enExample) |
| DK (2) | DK3821912T3 (enExample) |
| ES (2) | ES2834402T3 (enExample) |
| FI (1) | FI3821912T3 (enExample) |
| HR (2) | HRP20241154T1 (enExample) |
| HU (2) | HUE052781T2 (enExample) |
| LT (2) | LT3191139T (enExample) |
| PL (2) | PL3191139T3 (enExample) |
| PT (2) | PT3191139T (enExample) |
| RS (2) | RS61307B1 (enExample) |
| SI (2) | SI3191139T1 (enExample) |
| SM (2) | SMT202000715T1 (enExample) |
| WO (1) | WO2016014353A1 (enExample) |
Families Citing this family (9)
| 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 |
| US10646588B2 (en) | 2015-03-11 | 2020-05-12 | 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 |
| US11723988B2 (en) | 2017-10-20 | 2023-08-15 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector |
| WO2019099696A1 (en) * | 2017-11-15 | 2019-05-23 | The Regents Of The University Of Michigan | Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies |
| JP2023517929A (ja) | 2020-03-11 | 2023-04-27 | マサチューセッツ アイ アンド イヤー インファーマリー | Ｎｍｎａｔ１関連網膜変性に対する遺伝子療法 |
| 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)
| 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. |
| JP3015383B2 (ja) | 1987-09-11 | 2000-03-06 | 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 | 形質導入した線維芽およびそれらの使用 |
| DE3851153T2 (de) | 1987-12-11 | 1995-01-05 | Hughes Howard Med Inst | Genetische modifizierung von endothelialen zellen. |
| DE68927996T2 (de) | 1988-02-05 | 1997-12-04 | Hughes Howard Med Inst | Modifizierte hepatozyten und deren anwendung |
| ATE163046T1 (de) | 1990-10-31 | 1998-02-15 | Somatix Therapy Corp | Genetische veränderung von endothelzellen |
| 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 |
| JP6199965B2 (ja) * | 2012-07-11 | 2017-09-20 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Ｒｐｇｒｘ連鎖性網膜変性のａａｖ媒介型遺伝子治療 |
| 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 |
-
2015
- 2015-07-17 PT PT158253831T patent/PT3191139T/pt unknown
- 2015-07-17 CA CA2991750A patent/CA2991750C/en active Active
- 2015-07-17 HU HUE15825383A patent/HUE052781T2/hu unknown
- 2015-07-17 LT LTEP15825383.1T patent/LT3191139T/lt unknown
- 2015-07-17 PL PL15825383T patent/PL3191139T3/pl unknown
- 2015-07-17 HR HRP20241154TT patent/HRP20241154T1/hr unknown
- 2015-07-17 JP JP2017525514A patent/JP6654760B2/ja active Active
- 2015-07-17 SI SI201531413T patent/SI3191139T1/sl unknown
- 2015-07-17 HU HUE20199208A patent/HUE068183T2/hu unknown
- 2015-07-17 WO PCT/US2015/040866 patent/WO2016014353A1/en not_active Ceased
- 2015-07-17 FI FIEP20199208.8T patent/FI3821912T3/fi active
- 2015-07-17 ES ES15825383T patent/ES2834402T3/es active Active
- 2015-07-17 EP EP15825383.1A patent/EP3191139B1/en active Active
- 2015-07-17 RS RS20201416A patent/RS61307B1/sr unknown
- 2015-07-17 DK DK20199208.8T patent/DK3821912T3/da active
- 2015-07-17 ES ES20199208T patent/ES2987090T3/es active Active
- 2015-07-17 CN CN201580051512.3A patent/CN107206105A/zh active Pending
- 2015-07-17 US US15/328,617 patent/US10314924B2/en active Active
- 2015-07-17 EP EP20199208.8A patent/EP3821912B1/en active Active
- 2015-07-17 SM SM20200715T patent/SMT202000715T1/it unknown
- 2015-07-17 RS RS20240901A patent/RS65909B1/sr unknown
- 2015-07-17 DK DK15825383.1T patent/DK3191139T3/da active
- 2015-07-17 SI SI201532022T patent/SI3821912T1/sl unknown
- 2015-07-17 PT PT201992088T patent/PT3821912T/pt unknown
- 2015-07-17 PL PL20199208.8T patent/PL3821912T3/pl unknown
- 2015-07-17 SM SM20240309T patent/SMT202400309T1/it unknown
- 2015-07-17 LT LTEP20199208.8T patent/LT3821912T/lt unknown
- 2015-07-17 HR HRP20202023TT patent/HRP20202023T1/hr unknown
-
2019
- 2019-06-10 US US16/436,521 patent/US11045558B2/en active Active
- 2019-12-26 JP JP2019235539A patent/JP6966532B2/ja active Active
-
2021
- 2021-01-05 CY CY20211100002T patent/CY1123793T1/el unknown
- 2021-10-21 JP JP2021172128A patent/JP7198329B2/ja active Active
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