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
- 201000007737 Retinal degeneration Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 230000001404 mediated effect Effects 0.000 title 1
- 230000004258 retinal degeneration Effects 0.000 title 1
- 208000007014 Retinitis pigmentosa Diseases 0.000 abstract 2
- 201000004569 Blindness Diseases 0.000 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 101001104102 Homo sapiens X-linked retinitis pigmentosa GTPase regulator Proteins 0.000 abstract 1
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 1
- 101150059532 RPGR gene Proteins 0.000 abstract 1
- 102100040092 X-linked retinitis pigmentosa GTPase regulator Human genes 0.000 abstract 1
- 239000003937 drug carrier Substances 0.000 abstract 1
- 239000012634 fragment Substances 0.000 abstract 1
- 150000007523 nucleic acids Chemical group 0.000 abstract 1
- 210000000608 photoreceptor cell Anatomy 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 230000001105 regulatory effect Effects 0.000 abstract 1
- 230000004393 visual impairment Effects 0.000 abstract 1
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)
- Proteomics, Peptides & Aminoacids (AREA)
- Organic Chemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Psychology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Marine Sciences & Fisheries (AREA)
- Toxicology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (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 (enExample) |
| EP (1) | EP2872183B1 (enExample) |
| JP (1) | JP6199965B2 (enExample) |
| CN (1) | CN105120901A (enExample) |
| AU (1) | AU2013287281B2 (enExample) |
| CA (1) | CA2878171C (enExample) |
| NZ (1) | NZ704275A (enExample) |
| WO (1) | WO2014011210A1 (enExample) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2878171C (en) * | 2012-07-11 | 2021-04-27 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
| ES2760263T3 (es) * | 2014-03-21 | 2020-05-13 | Genzyme Corp | Tratamiento génico de la retinitis pigmentaria |
| JP6635942B2 (ja) * | 2014-04-15 | 2020-01-29 | アプライド ジェネティック テクノロジーズ コーポレイション | 網膜色素変性症ｇｔｐアーゼレギュレーター（ｒｐｇｒ）をコードするコドン最適化核酸 |
| 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 |
| EP3748007B1 (en) * | 2015-03-06 | 2024-06-26 | Massachusetts Eye & Ear Infirmary | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
| EP3719134B1 (en) * | 2015-03-11 | 2023-08-23 | The United States of America, as represented by The Secretary, Department of Health and Human Services | Rp2 vectors for treating x-linked retinitis pigmentosa |
| WO2016193434A1 (en) * | 2015-06-04 | 2016-12-08 | 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 |
| WO2017087900A1 (en) | 2015-11-19 | 2017-05-26 | The Trustees Of The University Of Pennsylvania | Compositions and methods for correction of heritable ocular disease |
| EP3389724B1 (en) | 2015-12-14 | 2020-07-01 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| CN109661470A (zh) | 2016-04-15 | 2019-04-19 | 宾夕法尼亚州大学信托人 | 新型aav8突变衣壳和含有其的组合物 |
| EP3481958A4 (en) | 2016-07-08 | 2019-12-25 | The Trustees of The University of Pennsylvania | METHOD AND COMPOSITIONS FOR TREATING DISTURBANCES AND DISEASES WITH RDH12 |
| CN119752819A (zh) | 2017-03-01 | 2025-04-04 | 宾夕法尼亚州立大学托管会 | 用于眼部病症的基因疗法 |
| WO2018200542A1 (en) | 2017-04-24 | 2018-11-01 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| WO2018218359A1 (en) | 2017-05-31 | 2018-12-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating peroxisomal disorders |
| EP3638316A4 (en) | 2017-06-14 | 2021-03-24 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR EYE DISEASES |
| AU2018367606B2 (en) * | 2017-11-15 | 2024-08-29 | The Regents Of The University Of Michigan | Viral vectors comprising RDH12 coding regions and methods of treating retinal dystrophies |
| KR102866133B1 (ko) | 2018-04-17 | 2025-09-30 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 트랜스-스플라이싱 분자 |
| WO2020061528A1 (en) | 2018-09-21 | 2020-03-26 | University Of Connecticut | Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome |
| CA3131023A1 (en) * | 2019-02-22 | 2020-08-27 | Michael R. Volkert | Oxr1 gene therapy |
| US20230139443A1 (en) * | 2020-02-28 | 2023-05-04 | The Trustees Of The University Of Pennsylvania | Treating autosomal dominant 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 |
| EP4208156A4 (en) | 2020-09-02 | 2025-01-15 | 4D Molecular Therapeutics Inc. | CODON-OPTIMIZED RPGRRF-15 GENES AND USES THEREOF |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2023220742A2 (en) | 2022-05-13 | 2023-11-16 | 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 |
| IL318200A (en) * | 2022-07-14 | 2025-03-01 | Fond Asile Des Aveugles | Gene therapy for FAM161A-related 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 |
| US5939534A (en) | 1993-12-29 | 1999-08-17 | Sumitomo Pharmaceuticals Company, Limited | Factors mutated in the D1 cap region |
| 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 |
| US6372250B1 (en) * | 2000-04-25 | 2002-04-16 | The Regents Of The University Of California | Non-invasive gene targeting to the brain |
| EP1381276A4 (en) | 2001-04-13 | 2005-02-02 | Univ Pennsylvania | METHOD FOR TREATMENT OR DEVELOPMENT SLUDGE DEGRADATION |
| US20030003582A1 (en) | 2001-05-08 | 2003-01-02 | Tranzyme, Inc. | Trans-viral vector mediated gene transfer to the retina |
| ES2602352T3 (es) | 2001-12-17 | 2017-02-20 | The Trustees Of The University Of Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas |
| DK2311848T3 (da) | 2002-12-23 | 2013-10-14 | Vical Inc | Kodon-optimerede polynukleotidbaserede vacciner mod human cytomegalovirus-infektion |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| 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 |
| CA2878171C (en) * | 2012-07-11 | 2021-04-27 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
-
2013
- 2013-01-23 CA CA2878171A patent/CA2878171C/en active Active
- 2013-01-23 NZ NZ704275A patent/NZ704275A/en unknown
- 2013-01-23 JP JP2015521593A patent/JP6199965B2/ja active Active
- 2013-01-23 WO PCT/US2013/022628 patent/WO2014011210A1/en not_active Ceased
- 2013-01-23 AU AU2013287281A patent/AU2013287281B2/en active Active
- 2013-01-23 US US14/413,884 patent/US9770491B2/en active Active
- 2013-01-23 CN CN201380045636.1A patent/CN105120901A/zh active Pending
- 2013-01-23 EP EP13816245.8A patent/EP2872183B1/en active Active
-
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 |
| US20150202269A1 (en) | 2015-07-23 |
| JP2015523379A (ja) | 2015-08-13 |
| US10383922B2 (en) | 2019-08-20 |
| AU2013287281A1 (en) | 2015-02-19 |
| CA2878171C (en) | 2021-04-27 |
| CN105120901A (zh) | 2015-12-02 |
| AU2013287281B2 (en) | 2018-04-26 |
| US9770491B2 (en) | 2017-09-26 |
| EP2872183A1 (en) | 2015-05-20 |
| JP6199965B2 (ja) | 2017-09-20 |
| EP2872183B1 (en) | 2018-09-26 |
| EP2872183A4 (en) | 2015-12-09 |
| US20180036385A1 (en) | 2018-02-08 |
| CA2878171A1 (en) | 2014-01-16 |
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