DK2675902T3 - Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel - Google Patents
Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel Download PDFInfo
- Publication number
- DK2675902T3 DK2675902T3 DK12705773.5T DK12705773T DK2675902T3 DK 2675902 T3 DK2675902 T3 DK 2675902T3 DK 12705773 T DK12705773 T DK 12705773T DK 2675902 T3 DK2675902 T3 DK 2675902T3
- Authority
- DK
- Denmark
- Prior art keywords
- aav9
- compositions
- improving
- methods
- tissue specificity
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- 239000000203 mixture Substances 0.000 title 1
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/66—Phosphorus compounds
- A61K31/683—Diesters of a phosphorus acid with two hydroxy compounds, e.g. phosphatidylinositols
- A61K31/685—Diesters of a phosphorus acid with two hydroxy compounds, e.g. phosphatidylinositols one of the hydroxy compounds having nitrogen atoms, e.g. phosphatidylserine, lecithin
-
- 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)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- 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
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- 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
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- 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
- C12N15/861—Adenoviral vectors
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- 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
- C12N2750/14041—Use of virus, viral particle or viral elements as a vector
- C12N2750/14043—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
- 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
- C12N2750/14041—Use of virus, viral particle or viral elements as a vector
- C12N2750/14045—Special targeting system for 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
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
- C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
- C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161443879P | 2011-02-17 | 2011-02-17 | |
| PCT/US2012/025550 WO2012112832A1 (en) | 2011-02-17 | 2012-02-17 | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2675902T3 true DK2675902T3 (da) | 2019-06-03 |
Family
ID=45757234
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK12705773.5T DK2675902T3 (da) | 2011-02-17 | 2012-02-17 | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel |
Country Status (16)
| Country | Link |
|---|---|
| US (5) | US9884071B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP2675902B1 (cg-RX-API-DMAC7.html) |
| JP (1) | JP6224459B2 (cg-RX-API-DMAC7.html) |
| KR (1) | KR101956885B1 (cg-RX-API-DMAC7.html) |
| CN (1) | CN103502458B (cg-RX-API-DMAC7.html) |
| AU (1) | AU2012219380B2 (cg-RX-API-DMAC7.html) |
| BR (1) | BR112013020734A2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2826316C (cg-RX-API-DMAC7.html) |
| DK (1) | DK2675902T3 (cg-RX-API-DMAC7.html) |
| ES (1) | ES2724800T3 (cg-RX-API-DMAC7.html) |
| HU (1) | HUE043921T2 (cg-RX-API-DMAC7.html) |
| MX (1) | MX353930B (cg-RX-API-DMAC7.html) |
| PL (1) | PL2675902T3 (cg-RX-API-DMAC7.html) |
| SG (3) | SG192268A1 (cg-RX-API-DMAC7.html) |
| TR (1) | TR201908020T4 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2012112832A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (121)
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| CN102199626B (zh) | 2003-09-30 | 2015-06-24 | 宾夕法尼亚大学托管会 | 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 |
| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| JP2013533847A (ja) | 2010-04-23 | 2013-08-29 | ユニバーシティ オブ マサチューセッツ | コレステロール関連障害のａａｖベースの治療 |
| CA2833908C (en) | 2010-04-23 | 2021-02-09 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| WO2012109570A1 (en) * | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| DK2675902T3 (da) | 2011-02-17 | 2019-06-03 | Univ Pennsylvania | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel |
| EP3318634A1 (en) | 2011-04-21 | 2018-05-09 | University of Massachusetts | Raav-based compositions and methods for treating diseases involving dominant-negative or gain of function mutations |
| JP6385920B2 (ja) * | 2012-05-09 | 2018-09-05 | オレゴン ヘルス アンド サイエンス ユニバーシティー | アデノ随伴ウイルスプラスミド及びベクター |
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| PT2968605T (pt) * | 2013-03-15 | 2022-09-22 | Univ North Carolina Chapel Hill | Métodos e composições para vetores aav de ligação dupla de glicano |
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| US10689653B2 (en) | 2014-06-03 | 2020-06-23 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| PT4012035T (pt) | 2014-09-16 | 2025-02-11 | Genzyme Corp | Vetores virais adenoassociados para tratar o glaucoma de miocilina (mioc) |
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| WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
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| EP2675902A1 (en) | 2013-12-25 |
| BR112013020734A2 (pt) | 2017-06-13 |
| US20210145857A1 (en) | 2021-05-20 |
| SG10201601110VA (en) | 2016-03-30 |
| KR20140046407A (ko) | 2014-04-18 |
| HUE043921T2 (hu) | 2019-09-30 |
| US20200101099A1 (en) | 2020-04-02 |
| AU2012219380B2 (en) | 2017-04-13 |
| US10406173B2 (en) | 2019-09-10 |
| MX353930B (es) | 2018-02-02 |
| US11766448B2 (en) | 2023-09-26 |
| JP6224459B2 (ja) | 2017-11-01 |
| US9884071B2 (en) | 2018-02-06 |
| US20240082288A1 (en) | 2024-03-14 |
| SG192268A1 (en) | 2013-08-30 |
| US20180185405A1 (en) | 2018-07-05 |
| SG10202007803QA (en) | 2020-09-29 |
| CA2826316C (en) | 2021-04-27 |
| EP2675902B1 (en) | 2019-03-27 |
| JP2014507154A (ja) | 2014-03-27 |
| ES2724800T3 (es) | 2019-09-16 |
| CN103502458A (zh) | 2014-01-08 |
| AU2012219380A1 (en) | 2013-08-29 |
| CA2826316A1 (en) | 2012-08-23 |
| TR201908020T4 (tr) | 2019-06-21 |
| US10918658B2 (en) | 2021-02-16 |
| PL2675902T3 (pl) | 2019-08-30 |
| US20130323226A1 (en) | 2013-12-05 |
| WO2012112832A1 (en) | 2012-08-23 |
| CN103502458B (zh) | 2016-11-16 |
| KR101956885B1 (ko) | 2019-03-12 |
| MX2013009538A (es) | 2013-09-26 |
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