DK2954051T3 - Modificeret kapsid til genoverførsel til behandling af nethinden - Google Patents
Modificeret kapsid til genoverførsel til behandling af nethinden Download PDFInfo
- Publication number
- DK2954051T3 DK2954051T3 DK14749120.3T DK14749120T DK2954051T3 DK 2954051 T3 DK2954051 T3 DK 2954051T3 DK 14749120 T DK14749120 T DK 14749120T DK 2954051 T3 DK2954051 T3 DK 2954051T3
- Authority
- DK
- Denmark
- Prior art keywords
- kapsid
- modified
- network
- transfer
- treatment
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
- C07K7/04—Linear peptides containing only normal peptide links
- C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
-
- 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
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361762775P | 2013-02-08 | 2013-02-08 | |
| PCT/US2014/015340 WO2014124282A1 (en) | 2013-02-08 | 2014-02-07 | Enhanced aav-mediated gene transfer for retinal therapies |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2954051T3 true DK2954051T3 (da) | 2019-07-08 |
Family
ID=51300160
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK14749120.3T DK2954051T3 (da) | 2013-02-08 | 2014-02-07 | Modificeret kapsid til genoverførsel til behandling af nethinden |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US9567376B2 (da) |
| EP (1) | EP2954051B1 (da) |
| DK (1) | DK2954051T3 (da) |
| ES (1) | ES2741502T3 (da) |
| WO (1) | WO2014124282A1 (da) |
Families Citing this family (79)
| Publication number | Priority date | Publication date | Assignee | Title |
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| CN101528916B (zh) | 2006-04-28 | 2013-09-04 | 宾夕法尼亚大学托管会 | 规模可调的aav生产方法 |
| SG194583A1 (en) | 2011-04-22 | 2013-12-30 | Univ California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| JP6600624B2 (ja) | 2013-05-31 | 2019-10-30 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | アデノ随伴ウイルス変異体及びその使用方法 |
| WO2015126972A1 (en) * | 2014-02-21 | 2015-08-27 | University Of Florida Research Foundation, Inc. | Methods and compositions for gene delivery to on bipolar cells |
| WO2015164778A1 (en) | 2014-04-25 | 2015-10-29 | The Trustees Of The University Of Pennysylvania | Ldlr variants and their use in compositions for reducing cholesterol levels |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CN112553229A (zh) | 2014-11-05 | 2021-03-26 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
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| US10472650B2 (en) | 2015-02-20 | 2019-11-12 | University Of Iowa Research Foundation | Methods and compositions for treating genetic eye diseases |
| KR20180055872A (ko) | 2015-09-24 | 2018-05-25 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 보체 매개 질환 치료용 조성물 및 방법 |
| EP3831842A1 (en) | 2015-09-28 | 2021-06-09 | The University of North Carolina at Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| US20180230489A1 (en) | 2015-10-28 | 2018-08-16 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| BR112018011687A2 (pt) | 2015-12-11 | 2018-12-04 | Univ Pennsylvania | terapia de gene para tratar hipercolesterolemia familiar |
| ES2918998T3 (es) | 2015-12-11 | 2022-07-21 | Univ Pennsylvania | Método de purificación escalable para AAVrh10 |
| US11015173B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV1 |
| AU2016370487C1 (en) | 2015-12-14 | 2022-03-03 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| CA3007330A1 (en) | 2015-12-14 | 2017-06-22 | The Trustees Of The University Of Pennsylvania | Composition for treatment of crigler-najjar syndrome |
| IL298604A (en) * | 2016-04-15 | 2023-01-01 | Univ Pennsylvania | Novel aav8 mutant capsids and preparations containing them |
| AU2017248731A1 (en) | 2016-04-15 | 2018-10-18 | The Trustees Of The University Of Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
| US11401527B2 (en) | 2016-04-17 | 2022-08-02 | The Trustees Of The University Of Pennsylvania | Compositions and methods useful for prophylaxis of organophosphates |
| WO2017189959A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
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| AU2017321488B2 (en) | 2016-08-30 | 2022-10-20 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| US11554147B2 (en) | 2017-02-20 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| CA3054136A1 (en) | 2017-03-01 | 2018-09-07 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US7056502B2 (en) * | 2000-04-28 | 2006-06-06 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids |
| BRPI0214119B8 (pt) | 2001-11-13 | 2021-05-25 | Univ Pennsylvania | vírus adenoassociado recombinante, método de geração do referido vírus e composição compreendendo o referido vírus |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| DE10234706B4 (de) | 2002-07-30 | 2006-06-08 | Siemens Ag | Verfahren zur Bestimmung der Kraftstoffmenge für eine Brennkraftmaschine |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| ES2538468T3 (es) | 2008-05-20 | 2015-06-22 | Eos Neuroscience, Inc. | Vectores para la administración de proteínas sensibles a la luz y métodos para su utilización |
| EP2287323A1 (en) | 2009-07-31 | 2011-02-23 | Association Institut de Myologie | Widespread gene delivery to the retina using systemic administration of AAV vectors |
| US20140099284A1 (en) | 2010-10-15 | 2014-04-10 | Eos Neuroscience, Inc | Modulation neural pathways |
| SG194583A1 (en) | 2011-04-22 | 2013-12-30 | Univ California | Adeno-associated virus virions with variant capsid and methods of use thereof |
-
2014
- 2014-02-07 ES ES14749120T patent/ES2741502T3/es active Active
- 2014-02-07 EP EP14749120.3A patent/EP2954051B1/en active Active
- 2014-02-07 US US14/766,172 patent/US9567376B2/en active Active
- 2014-02-07 DK DK14749120.3T patent/DK2954051T3/da active
- 2014-02-07 WO PCT/US2014/015340 patent/WO2014124282A1/en active Application Filing
Also Published As
| Publication number | Publication date |
|---|---|
| WO2014124282A1 (en) | 2014-08-14 |
| ES2741502T3 (es) | 2020-02-11 |
| US20150376240A1 (en) | 2015-12-31 |
| US9567376B2 (en) | 2017-02-14 |
| EP2954051A4 (en) | 2016-09-14 |
| EP2954051A1 (en) | 2015-12-16 |
| EP2954051B1 (en) | 2019-03-27 |
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| DK3084009T3 (da) | Lactobacillus salivarius til behandling af mastitis | |
| DK3038656T3 (da) | Forbindelse af glycosaminoglycan, fremgangsmåde til fremstilling og anvendelse deraf | |
| DK3063264T3 (da) | Tørring af mikroorganismer | |
| MA41266A (fr) | Cataplasme | |
| DK2932944T3 (da) | Støtteortose | |
| DK3052931T3 (da) | Fremgangsmåde til overførsel af opslæmning |