DK2425000T3 - Sammensætninger rettet mod ledende luftvejsceller omfattende konstrukter af adeno-associeret virus - Google Patents
Sammensætninger rettet mod ledende luftvejsceller omfattende konstrukter af adeno-associeret virus Download PDFInfo
- Publication number
- DK2425000T3 DK2425000T3 DK10716723.1T DK10716723T DK2425000T3 DK 2425000 T3 DK2425000 T3 DK 2425000T3 DK 10716723 T DK10716723 T DK 10716723T DK 2425000 T3 DK2425000 T3 DK 2425000T3
- Authority
- DK
- Denmark
- Prior art keywords
- associated viruses
- compositions related
- adeno associated
- leading aircraft
- including adeno
- Prior art date
Links
- 241000700605 Viruses Species 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
Classifications
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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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- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
- A61P11/06—Antiasthmatics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/12—Antihypertensives
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
-
- 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
- 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
- C12N2750/14145—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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Biotechnology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Pulmonology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US17414909P | 2009-04-30 | 2009-04-30 | |
| PCT/US2010/032943 WO2010127097A1 (en) | 2009-04-30 | 2010-04-29 | Compositions for targeting conducting airway cells comprising adeno-associated virus constructs |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2425000T3 true DK2425000T3 (da) | 2019-05-13 |
Family
ID=42358394
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK10716723.1T DK2425000T3 (da) | 2009-04-30 | 2010-04-29 | Sammensætninger rettet mod ledende luftvejsceller omfattende konstrukter af adeno-associeret virus |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US10920244B2 (da) |
| EP (1) | EP2425000B1 (da) |
| JP (2) | JP6174318B2 (da) |
| CN (1) | CN102439157B (da) |
| DK (1) | DK2425000T3 (da) |
| ES (1) | ES2724122T3 (da) |
| HU (1) | HUE044522T2 (da) |
| PL (1) | PL2425000T3 (da) |
| TR (1) | TR201906398T4 (da) |
| WO (1) | WO2010127097A1 (da) |
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| SG10202007803QA (en) | 2011-02-17 | 2020-09-29 | Univ Pennsylvania | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
| DE102013215817A1 (de) | 2013-08-09 | 2015-02-12 | Universitätsklinikum Hamburg-Eppendorf | Neue peptide mit spezifität für die lunge |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| DE102014207498A1 (de) | 2014-04-17 | 2015-10-22 | Universitätsklinikum Hamburg-Eppendorf | Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
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| WO2025113676A1 (en) | 2023-11-29 | 2025-06-05 | Neuexcell Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treating stroke in primates |
| WO2025217214A2 (en) | 2024-04-08 | 2025-10-16 | Regenxbio Inc. | Recombinant adeno-associated viruses and uses thereof |
| WO2025217230A1 (en) | 2024-04-08 | 2025-10-16 | Regenxbio Inc. | Vectorized anti-complement antibodies and complement agents and administration thereof |
| WO2025237990A1 (en) | 2024-05-14 | 2025-11-20 | Institut National de la Santé et de la Recherche Médicale | Antisense oligonucleotides and their use for the treatment of pulmonary fibrosis |
| WO2026006341A1 (en) | 2024-06-24 | 2026-01-02 | Regenxbio Inc. | Microdystrophin gene therapy administration for treatment of dystrophinopathies |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| JP2006075171A (ja) * | 1995-11-09 | 2006-03-23 | Avigen Inc | 組換えaavビリオン産生における使用のための補助機能 |
| US6004797A (en) | 1995-11-09 | 1999-12-21 | Avigen, Inc. | Adenovirus helper-free recombinant AAV Virion production |
| JP2001500015A (ja) | 1996-09-06 | 2001-01-09 | 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 | T7ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法 |
| JP2001517454A (ja) | 1997-09-19 | 2001-10-09 | 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 | 組換えアデノ随伴ウイルスの産生に有用な方法および細胞株 |
| ATE454445T1 (de) * | 1998-11-10 | 2010-01-15 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| SG10202108118RA (en) | 2001-11-13 | 2021-08-30 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| US8927269B2 (en) * | 2003-05-19 | 2015-01-06 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Avian adenoassociated virus and uses thereof |
| HUE054805T2 (hu) * | 2003-09-30 | 2021-09-28 | Univ Pennsylvania | Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik |
| EP3085389A1 (en) * | 2005-04-07 | 2016-10-26 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| CN101495624A (zh) | 2006-04-28 | 2009-07-29 | 宾夕法尼亚州立大学托管会 | 衣壳免疫原性降低的经修饰aav载体及其用途 |
| WO2009137006A2 (en) * | 2008-04-30 | 2009-11-12 | The University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
| JP5791047B2 (ja) | 2009-03-26 | 2015-10-07 | 国立大学法人東京海洋大学 | コイヘルペスウイルス特異的抗体及びその抗原 |
| ES2724122T3 (es) * | 2009-04-30 | 2019-09-06 | Univ Pennsylvania | Composiciones para dirigir células de las vías respiratorias de conducción que comprenden construcciones de virus adenoasociado |
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2010
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- 2010-04-29 JP JP2012508722A patent/JP6174318B2/ja not_active Expired - Fee Related
- 2010-04-29 HU HUE10716723 patent/HUE044522T2/hu unknown
- 2010-04-29 TR TR2019/06398T patent/TR201906398T4/tr unknown
- 2010-04-29 US US13/266,599 patent/US10920244B2/en not_active Expired - Fee Related
- 2010-04-29 EP EP10716723.1A patent/EP2425000B1/en active Active
- 2010-04-29 WO PCT/US2010/032943 patent/WO2010127097A1/en not_active Ceased
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- 2010-04-29 PL PL10716723T patent/PL2425000T3/pl unknown
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2017
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2021
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Also Published As
| Publication number | Publication date |
|---|---|
| JP2012525150A (ja) | 2012-10-22 |
| US20210147875A1 (en) | 2021-05-20 |
| TR201906398T4 (tr) | 2019-05-21 |
| HUE044522T2 (hu) | 2019-10-28 |
| ES2724122T3 (es) | 2019-09-06 |
| JP2017127316A (ja) | 2017-07-27 |
| US20120046349A1 (en) | 2012-02-23 |
| PL2425000T3 (pl) | 2019-08-30 |
| CN102439157B (zh) | 2015-09-16 |
| JP6174318B2 (ja) | 2017-08-02 |
| EP2425000A1 (en) | 2012-03-07 |
| CN102439157A (zh) | 2012-05-02 |
| US10920244B2 (en) | 2021-02-16 |
| EP2425000B1 (en) | 2019-02-20 |
| WO2010127097A1 (en) | 2010-11-04 |
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