NZ704275A - Aav-mediated gene therapy for rpgr x-linked retinal degeneration - Google Patents
Aav-mediated gene therapy for rpgr x-linked retinal degenerationInfo
- Publication number
- NZ704275A NZ704275A NZ704275A NZ70427513A NZ704275A NZ 704275 A NZ704275 A NZ 704275A NZ 704275 A NZ704275 A NZ 704275A NZ 70427513 A NZ70427513 A NZ 70427513A NZ 704275 A NZ704275 A NZ 704275A
- Authority
- NZ
- New Zealand
- Prior art keywords
- aav
- subject
- rpgr
- retinitis pigmentosa
- gene therapy
- Prior art date
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/18—Growth factors; Growth regulators
- A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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
-
- 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
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
- C12Y306/05—Hydrolases acting on acid anhydrides (3.6) acting on GTP; involved in cellular and subcellular movement (3.6.5)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Psychology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Marine Sciences & Fisheries (AREA)
- Toxicology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Ophthalmology & Optometry (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261670355P | 2012-07-11 | 2012-07-11 | |
| PCT/US2013/022628 WO2014011210A1 (en) | 2012-07-11 | 2013-01-23 | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| NZ704275A true NZ704275A (en) | 2016-09-30 |
Family
ID=49916455
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| NZ704275A NZ704275A (en) | 2012-07-11 | 2013-01-23 | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US9770491B2 (https=) |
| EP (1) | EP2872183B1 (https=) |
| JP (1) | JP6199965B2 (https=) |
| CN (1) | CN105120901A (https=) |
| AU (1) | AU2013287281B2 (https=) |
| CA (1) | CA2878171C (https=) |
| NZ (1) | NZ704275A (https=) |
| WO (1) | WO2014011210A1 (https=) |
Families Citing this family (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| FI3628334T3 (fi) | 2014-03-21 | 2023-09-15 | Genzyme Corp | Geenihoito verkkokalvon pigmenttirappeuma |
| CA2945965C (en) * | 2014-04-15 | 2022-11-01 | Applied Genetic Technologies Corporation | Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (rpgr) |
| 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 |
| PL3265568T3 (pl) * | 2015-03-06 | 2020-11-30 | Massachusetts Eye & Ear Infirmary | Terapie augmentacji genów w przypadku wrodzonego zwyrodnienia siatkówki spowodowanego mutacjami genu prpf31 |
| 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 |
| US20180161405A1 (en) * | 2015-06-04 | 2018-06-14 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Improved methods for treating ocular diseases by gene therapy |
| GB201516066D0 (en) | 2015-09-10 | 2015-10-28 | Young & Co Llp D | Treatment of retinitis pigmentosa |
| EP3377116A4 (en) | 2015-11-19 | 2019-07-10 | The Trustees of The University of Pennsylvania | COMPOSITIONS AND METHODS FOR CORRECTING ECG-RELATED EYE DISEASE |
| US11090392B2 (en) | 2015-12-14 | 2021-08-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| WO2017180854A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Novel aav8 mutant capsids and compositions containing same |
| US11197936B2 (en) | 2016-07-08 | 2021-12-14 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treatment of disorders and diseases involving RDH12 |
| BR112019017327A2 (pt) | 2017-03-01 | 2020-04-14 | Univ Pennsylvania | terapia gênica para distúrbios oculares |
| WO2018200542A1 (en) | 2017-04-24 | 2018-11-01 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| EP3630986B1 (en) | 2017-05-31 | 2026-03-25 | The Trustees of The University of Pennsylvania | Gene therapy for treating peroxisomal disorders |
| US11827898B2 (en) | 2017-06-14 | 2023-11-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| KR20200036912A (ko) | 2017-07-31 | 2020-04-07 | 리플렉션 바이오테크놀러지스 리미티드 | 안과 질환을 위한 세포 모델 및 치료요법 |
| SG11202004527YA (en) * | 2017-11-15 | 2020-06-29 | Univ Michigan Regents | Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies |
| CA3097004A1 (en) | 2018-04-17 | 2019-10-24 | The Trustees Of The University Of Pennsylvania | Trans-splicing molecules |
| CA3113648A1 (en) | 2018-09-21 | 2020-03-26 | University Of Connecticut | Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome |
| US20220054655A1 (en) * | 2019-02-22 | 2022-02-24 | University Of Massachusetts | Oxr1 gene therapy |
| EP3934699A4 (en) | 2019-03-04 | 2022-12-21 | The Trustees of The University of Pennsylvania | NEUROPROTECTIVE GENE THERAPY TARGETING THE AKT PATHWAY |
| US20230112568A1 (en) * | 2020-02-28 | 2023-04-13 | The Trustees Of The University Of Pennsylvania | Treating autosomal recessive bestrophinopathies and methods for evaluating same |
| CN113952472A (zh) * | 2020-07-21 | 2022-01-21 | 英斯培瑞有限公司 | 用于治疗眼部疾病的组合物和方法 |
| US11345930B2 (en) | 2020-09-02 | 2022-05-31 | 4D Molecular Therapeutics Inc. | Codon optimized RPGRORF15 genes and uses thereof |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| AU2023269865A1 (en) | 2022-05-13 | 2024-11-21 | Ascidian Therapeutics, Inc. | Abca4 trans-splicing molecules |
| 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 |
| US20260000786A1 (en) * | 2022-07-14 | 2026-01-01 | Fondation Asile Des Aveugles | Gene therapy for fam161a-associated retinopathies and other ciliopathies |
| US12252518B2 (en) | 2023-01-06 | 2025-03-18 | Life Biosciences, Inc. | Methods of treating non-arteritic anterior ischemic optic neuropathy |
| CN117050951B (zh) * | 2023-08-24 | 2024-06-04 | 广州医药研究总院有限公司 | X连锁视网膜色素变性模型犬的构建方法 |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| JP3768257B2 (ja) * | 1993-12-29 | 2006-04-19 | 大日本住友製薬株式会社 | 改変タンパク質 |
| EP0749980A4 (en) | 1993-12-29 | 1999-09-29 | Sumitomo Pharma | NEW CELLAR FACTOR FROM HUMAN |
| US6372250B1 (en) * | 2000-04-25 | 2002-04-16 | The Regents Of The University Of California | Non-invasive gene targeting to the brain |
| CA2442670A1 (en) * | 2001-04-13 | 2002-10-24 | The Trustees Of The University Of Pennsylvania | Method of treating or retarding the development of blindness |
| US20030003582A1 (en) | 2001-05-08 | 2003-01-02 | Tranzyme, Inc. | Trans-viral vector mediated gene transfer to the retina |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| WO2004058166A2 (en) | 2002-12-23 | 2004-07-15 | Vical Incorporated | Codon-optimized polynucleotide-based vaccines against human cytomegalovirus infection |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| ES2331342B1 (es) | 2006-05-22 | 2010-10-13 | Consejo Superior Investg.Cientificas | Uso de la proinsulina para la elaboracion de una composicion farmaceutica neuroprotectora, composicion terapeutica que la contiene y sus aplicaciones. |
| AU2009212309B2 (en) | 2008-02-07 | 2014-04-03 | Ceregene, Inc. | Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein |
| US20100081707A1 (en) * | 2008-02-21 | 2010-04-01 | Ali Robin R | Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea |
| US7561973B1 (en) | 2008-07-31 | 2009-07-14 | Dna Twopointo, Inc. | Methods for determining properties that affect an expression property value of polynucleotides in an expression system |
| US7561972B1 (en) | 2008-06-06 | 2009-07-14 | Dna Twopointo, Inc. | Synthetic nucleic acids for expression of encoded proteins |
| ITMI20090284A1 (it) | 2009-02-26 | 2010-08-27 | Consiglio Nazionale Ricerche | Uso di inibitori della serina palmitoiltrasferasi per la prevenzione e il rallentamento delle degenerazioni retiniche ereditarie e relative composizioni |
| GB0911870D0 (en) * | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| 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 |
-
2013
- 2013-01-23 EP EP13816245.8A patent/EP2872183B1/en active Active
- 2013-01-23 AU AU2013287281A patent/AU2013287281B2/en active Active
- 2013-01-23 JP JP2015521593A patent/JP6199965B2/ja active Active
- 2013-01-23 NZ NZ704275A patent/NZ704275A/en unknown
- 2013-01-23 US US14/413,884 patent/US9770491B2/en active Active
- 2013-01-23 CA CA2878171A patent/CA2878171C/en active Active
- 2013-01-23 WO PCT/US2013/022628 patent/WO2014011210A1/en not_active Ceased
- 2013-01-23 CN CN201380045636.1A patent/CN105120901A/zh active Pending
-
2017
- 2017-09-08 US US15/699,262 patent/US10383922B2/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| WO2014011210A1 (en) | 2014-01-16 |
| US20180036385A1 (en) | 2018-02-08 |
| JP2015523379A (ja) | 2015-08-13 |
| EP2872183B1 (en) | 2018-09-26 |
| EP2872183A4 (en) | 2015-12-09 |
| US10383922B2 (en) | 2019-08-20 |
| CA2878171C (en) | 2021-04-27 |
| US20150202269A1 (en) | 2015-07-23 |
| AU2013287281A1 (en) | 2015-02-19 |
| US9770491B2 (en) | 2017-09-26 |
| JP6199965B2 (ja) | 2017-09-20 |
| CN105120901A (zh) | 2015-12-02 |
| EP2872183A1 (en) | 2015-05-20 |
| CA2878171A1 (en) | 2014-01-16 |
| AU2013287281B2 (en) | 2018-04-26 |
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