ES2711349T3 - Cápside - Google Patents
Cápside Download PDFInfo
- Publication number
- ES2711349T3 ES2711349T3 ES16721917T ES16721917T ES2711349T3 ES 2711349 T3 ES2711349 T3 ES 2711349T3 ES 16721917 T ES16721917 T ES 16721917T ES 16721917 T ES16721917 T ES 16721917T ES 2711349 T3 ES2711349 T3 ES 2711349T3
- Authority
- ES
- Spain
- Prior art keywords
- aav
- sequence
- sequences
- capsid
- seq
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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- NBQNWMBBSKPBAY-UHFFFAOYSA-N iodixanol Chemical compound IC=1C(C(=O)NCC(O)CO)=C(I)C(C(=O)NCC(O)CO)=C(I)C=1N(C(=O)C)CC(O)CN(C(C)=O)C1=C(I)C(C(=O)NCC(O)CO)=C(I)C(C(=O)NCC(O)CO)=C1I NBQNWMBBSKPBAY-UHFFFAOYSA-N 0.000 description 1
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Classifications
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
-
- 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
- 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
- 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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biophysics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB1508026.0A GB201508026D0 (en) | 2015-05-11 | 2015-05-11 | Capsid |
| PCT/GB2016/051329 WO2016181123A1 (en) | 2015-05-11 | 2016-05-10 | Capsid |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ES2711349T3 true ES2711349T3 (es) | 2019-05-03 |
Family
ID=53489438
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES16721917T Active ES2711349T3 (es) | 2015-05-11 | 2016-05-10 | Cápside |
Country Status (18)
| Country | Link |
|---|---|
| US (3) | US10414803B2 (enExample) |
| EP (2) | EP3250239B1 (enExample) |
| JP (2) | JP6619454B2 (enExample) |
| KR (1) | KR20180019543A (enExample) |
| CN (1) | CN107864653B (enExample) |
| AU (1) | AU2016261454B2 (enExample) |
| CA (1) | CA2985945C (enExample) |
| DK (1) | DK3250239T3 (enExample) |
| ES (1) | ES2711349T3 (enExample) |
| GB (1) | GB201508026D0 (enExample) |
| HR (1) | HRP20190442T1 (enExample) |
| HU (1) | HUE042693T2 (enExample) |
| LT (1) | LT3250239T (enExample) |
| PL (1) | PL3250239T3 (enExample) |
| PT (1) | PT3250239T (enExample) |
| TR (1) | TR201901582T4 (enExample) |
| TW (1) | TW201704253A (enExample) |
| WO (1) | WO2016181123A1 (enExample) |
Families Citing this family (47)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| GB201508026D0 (en) * | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| JP7064214B2 (ja) | 2015-09-28 | 2022-05-10 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 抗体回避性ウイルスベクターのための方法および組成物 |
| GB201608046D0 (en) | 2016-05-09 | 2016-06-22 | Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The | Treatment of complement-mediated disorders |
| MX2019007873A (es) | 2016-12-30 | 2019-11-18 | Univ Pennsylvania | Terapia génica para tratar la enfermedad de wilson. |
| EP3645021A4 (en) * | 2017-06-30 | 2021-04-21 | Intima Bioscience, Inc. | ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY |
| US11608510B2 (en) * | 2018-03-30 | 2023-03-21 | The Board Of Trustees Of The Leland Stanford Junior University | Recombinant adeno-associated virus capsids with enhanced human pancreatic tropism |
| MX2020010464A (es) | 2018-04-03 | 2021-01-29 | Vectores de virus que evitan anticuerpos. | |
| JP7425738B2 (ja) | 2018-04-03 | 2024-01-31 | ギンコ バイオワークス インコーポレイテッド | 眼組織を標的とするウイルスベクター |
| US12091435B2 (en) | 2018-04-03 | 2024-09-17 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| KR102114249B1 (ko) | 2018-08-21 | 2020-05-22 | 코나아이 (주) | 가상 화폐 결제 방법 및 시스템 |
| GB201817810D0 (en) | 2018-10-31 | 2018-12-19 | Ucl Business Plc | Modified factor IX polypeptides |
| PL3906066T3 (pl) | 2019-01-04 | 2024-09-02 | Ultragenyx Pharmaceutical Inc. | Konstrukty do terapii genowej do leczenia choroby wilsona |
| JP7698583B2 (ja) * | 2019-03-21 | 2025-06-25 | ギンコ バイオワークス インコーポレイテッド | 組換えアデノ随伴ウイルスベクター |
| EP3715358A1 (en) * | 2019-03-28 | 2020-09-30 | Universität zu Köln | Mutated adeno-associated virus capsid proteins, aav particle comprising the same and liver directed aav vector gene therapy |
| PT3722434T (pt) | 2019-04-12 | 2022-10-18 | Freeline Therapeutics Ltd | Sistema plasmídico |
| BR112021020401A2 (pt) | 2019-04-12 | 2021-12-07 | Freeline Therapeutics Ltd | Sistema de dois plasmídeos, plasmídeo auxiliar, plasmídeo vetor, usos do sistema de dois plasmídeos, métodos para controlar ou maximizar a razão de partículas completas para totais produzidas durante a produção de aav recombinante e para aumentar, otimizar ou maximizar o rendimento de aav recombinante |
| WO2021009805A1 (ja) * | 2019-07-12 | 2021-01-21 | 株式会社遺伝子治療研究所 | ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| WO2021030764A1 (en) * | 2019-08-14 | 2021-02-18 | University Of Florida Research Foundation, Incorporated | Aav capsid variants for gene therapy |
| JP2022545534A (ja) * | 2019-08-29 | 2022-10-27 | ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー | ヒト血液脳関門を通過するためのアデノ随伴ウイルスベクター |
| IL292297A (en) | 2019-10-17 | 2022-06-01 | Stridebio Inc | Adeno-associated viral vectors for treatment of niemann-pick disease type c |
| TW202142554A (zh) * | 2020-02-25 | 2021-11-16 | 澳洲兒童醫學研究所 | 腺相關病毒蛋白殼多肽及載體 |
| WO2022003211A1 (en) * | 2020-07-03 | 2022-01-06 | Genethon | Method for engineering novel hybrid aav capsids through hypervariable regions swapping |
| CN116121274B (zh) * | 2020-07-29 | 2024-11-19 | 北京三诺佳邑生物技术有限责任公司 | 一组肝靶向新型腺相关病毒的获得及其应用 |
| CA3189878A1 (en) | 2020-08-19 | 2022-02-24 | Colin O'BANION | Adeno-associated virus vectors for treatment of rett syndrome |
| WO2022060915A1 (en) | 2020-09-15 | 2022-03-24 | Regenxbio Inc. | Vectorized lanadelumab and administration thereof |
| EP4214242A1 (en) | 2020-09-15 | 2023-07-26 | RegenxBio Inc. | Vectorized antibodies for anti-viral therapy |
| GB202016254D0 (en) | 2020-10-13 | 2020-11-25 | Freeline Therapeutics Ltd | Plasmid system |
| WO2022094157A1 (en) | 2020-10-28 | 2022-05-05 | Regenxbio Inc. | Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof |
| TW202233841A (zh) | 2020-10-28 | 2022-09-01 | 美商銳進科斯生物股份有限公司 | 用於眼適應症之載體化抗ＴＮＦ－α抗體 |
| EP4236974A2 (en) | 2020-10-29 | 2023-09-06 | RegenxBio Inc. | Vectorized factor xii antibodies and administration thereof |
| CN113121654B (zh) * | 2021-04-19 | 2023-11-17 | 信念医药科技(上海)有限公司 | 腺相关病毒衣壳蛋白及包含其的腺相关病毒载体 |
| WO2022226301A1 (en) * | 2021-04-23 | 2022-10-27 | The University Of North Carolina At Chapel Hill | Chimeric heart tropic aav capsids |
| GB202109231D0 (en) | 2021-06-25 | 2021-08-11 | Freeline Therapeutics Ltd | Promoter |
| CN118201947B (zh) * | 2021-10-28 | 2025-03-21 | 上海天泽云泰生物医药有限公司 | 腺相关病毒衣壳 |
| GB202205514D0 (en) | 2022-04-13 | 2022-05-25 | Freeline Therapeutics Ltd | Mechanical lysis |
| TW202400803A (zh) | 2022-05-03 | 2024-01-01 | 美商銳進科斯生物股份有限公司 | 載體化抗補體抗體與補體劑及其投與 |
| US20250288697A1 (en) | 2022-05-03 | 2025-09-18 | Regenxbio Inc. | Vectorized anti-tnf-alpha inhibitors for ocular indications |
| GB202214328D0 (en) | 2022-09-29 | 2022-11-16 | Freeline Therapeutics Ltd | Method |
| WO2024238859A1 (en) | 2023-05-16 | 2024-11-21 | Regenxbio Inc. | Vectorized c5 inhibitor agents and administration thereof |
| AU2024271004A1 (en) | 2023-05-16 | 2026-01-15 | Regenxbio Inc. | Vectorized anti-complement antibodies and administration thereof |
| WO2025087695A1 (en) | 2023-10-23 | 2025-05-01 | Ascend Advanced Therapies Ltd | Method for producing recombinant adeno-associated virus particles |
| EP4628576A1 (en) | 2024-04-07 | 2025-10-08 | Ascend Advanced Therapies GmbH | Microrna molecules for use in methods for improving recombinant aav production |
| TW202548025A (zh) | 2024-04-08 | 2025-12-16 | 美商銳進科斯生物股份有限公司 | 載體化抗補體抗體與補體劑及其投與 |
| WO2025238067A1 (en) | 2024-05-15 | 2025-11-20 | Ascend Advanced Therapies Gmbh | Novel vector system for aav production |
Family Cites Families (101)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| EP1690868A1 (en) | 1997-10-31 | 2006-08-16 | Maxygen, Inc. | Modification of virus tropism and host range by viral genome shuffling |
| ATE454445T1 (de) | 1998-11-10 | 2010-01-15 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| EP1026250A1 (en) | 1998-12-02 | 2000-08-09 | Aventis Behring GmbH | DNA-constructs of blood clotting factors and P-selectin |
| EP1010762A1 (en) | 1998-12-02 | 2000-06-21 | Aventis Behring Gesellschaft mit beschränkter Haftung | DNA constructs of blood clotting factors and P-Selectin |
| EP1038959A1 (en) | 1999-03-17 | 2000-09-27 | Aventis Behring Gesellschaft mit beschränkter Haftung | Factor VIII without B-domain, comprising one or more insertions of a truncated intron I of factor IX |
| EP1048726B1 (en) | 1999-04-27 | 2006-07-26 | Négrier, Claude | Modified factor VIII cDNA |
| US20020045264A1 (en) | 2000-03-14 | 2002-04-18 | During Matthew J. | Production of chimeric capsid vectors |
| WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Aav2 vectors and methods |
| US7749492B2 (en) | 2001-01-05 | 2010-07-06 | Nationwide Children's Hospital, Inc. | AAV vectors and methods |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| CN103555677B (zh) | 2001-11-13 | 2018-01-30 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| WO2003052051A2 (en) | 2001-12-17 | 2003-06-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences |
| US7858367B2 (en) | 2002-04-30 | 2010-12-28 | Duke University | Viral vectors and methods for producing and using the same |
| JP2007524386A (ja) | 2003-06-19 | 2007-08-30 | アビジェン， インコーポレイテッド | 減少した免疫反応性を有するａａｖビリオン、およびその使用 |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
| WO2005072364A2 (en) | 2004-01-27 | 2005-08-11 | University Of Florida | A modified baculovirus expression system for production of pseudotyped raav vector |
| US7892809B2 (en) | 2004-12-15 | 2011-02-22 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| WO2006130639A2 (en) | 2005-05-31 | 2006-12-07 | Neurologix, Inc. | Novel glutamic acid decarboxylase (gad) chimera and methods of use |
| WO2007089632A2 (en) | 2006-01-27 | 2007-08-09 | The University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| MX2009001875A (es) | 2006-08-21 | 2009-03-02 | Genentech Inc | Compuestos de aza-benzotiofenilo y metodos de uso de los mismos. |
| US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| EP2623605B1 (en) | 2007-04-09 | 2018-11-28 | University of Florida Research Foundation, Inc. | RAAV vector compositions having tyrosine-modified capsid proteins and methods for use |
| US20100203083A1 (en) * | 2007-05-31 | 2010-08-12 | Medigene Ag | Mutated structural protein of a parvovirus |
| US8632764B2 (en) | 2008-04-30 | 2014-01-21 | University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
| EP2396343B1 (en) | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| US9464119B2 (en) | 2009-03-04 | 2016-10-11 | Deutsches Krebsforschungszentrum | Assembly activating protein (AAP) and its use for the manufacture of parvovirus particles essentially consisting of VP3 |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| US9169299B2 (en) | 2011-08-24 | 2015-10-27 | The Board Of Trustees Of The Leleand Stanford Junior University | AAV capsid proteins for nucleic acid transfer |
| CN104520421B (zh) | 2011-10-28 | 2017-05-24 | 北卡罗来纳-查佩尔山大学 | 用于生产腺伴随病毒的细胞系 |
| EP2900686B1 (en) | 2012-09-28 | 2020-06-10 | The University of North Carolina At Chapel Hill | Aav vectors targeted to oligodendrocytes |
| DK2911687T3 (da) | 2012-10-26 | 2019-05-13 | Univ Brussel Vrije | Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf |
| US10385112B2 (en) | 2013-03-14 | 2019-08-20 | Asklepios Biopharmaceutical, Inc. | Modified soluble VEGF receptor-1 genes and vectors for gene therapy |
| CN105163764B (zh) | 2013-03-15 | 2019-11-12 | 北卡罗来纳-查佩尔山大学 | 双重聚糖结合aav载体的方法和组合物 |
| EP2999791B1 (en) | 2013-05-21 | 2018-12-05 | University of Florida Research Foundation, Inc. | Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer |
| CA3209883A1 (en) | 2013-07-22 | 2015-01-29 | The Children's Hospital Of Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| LT3702466T (lt) | 2013-08-27 | 2023-02-27 | Research Institute At Nationwide Children's Hospital | Produktai ir būdai, skirti šoninės amiotrofinės sklerozės gydymui |
| ES2857751T3 (es) | 2013-10-11 | 2021-09-29 | Massachusetts Eye & Ear Infirmary | Métodos para predecir secuencias de virus ancestrales y usos de los mismos |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| WO2015126972A1 (en) | 2014-02-21 | 2015-08-27 | University Of Florida Research Foundation, Inc. | Methods and compositions for gene delivery to on bipolar cells |
| US10308957B2 (en) | 2014-03-04 | 2019-06-04 | University Of Florida Research Foundation, Inc. | rAAV vectors and methods for transduction of photoreceptors and RPE cells |
| DK3546585T3 (da) | 2014-04-25 | 2025-11-03 | Genethon | Nukleinsyrer med en modificeret intron til brug i behandlingen af hyperbilirubinæmi |
| WO2015168666A2 (en) | 2014-05-02 | 2015-11-05 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| WO2016065001A1 (en) | 2014-10-21 | 2016-04-28 | University Of Massachusetts | Recombinant aav variants and uses thereof |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| BR112017010447A2 (pt) | 2014-11-21 | 2018-05-15 | The University Of North Carolina At Chapel Hill | vetores de aav direcionados ao sistema nervoso central |
| GB201502137D0 (en) | 2015-02-09 | 2015-03-25 | Ucl Business Plc | Treatment |
| WO2016133917A1 (en) | 2015-02-16 | 2016-08-25 | University Of Florida Research Foundation | Raav vector compositions, methods for targeting vascular endothelial cells and use in treatment of type i diabetes |
| US20180245073A1 (en) | 2015-02-23 | 2018-08-30 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
| CN114480502A (zh) | 2015-03-02 | 2022-05-13 | 阿德夫拉姆生物技术股份有限公司 | 用于将多核苷酸玻璃体内递送到视网膜视锥的组合物和方法 |
| WO2016154344A1 (en) | 2015-03-24 | 2016-09-29 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| US10081659B2 (en) | 2015-04-06 | 2018-09-25 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Adeno-associated vectors for enhanced transduction and reduced immunogenicity |
| EP4491733A3 (en) | 2015-04-23 | 2025-03-26 | University of Massachusetts | Modulation of aav vector transgene expression |
| GB201508026D0 (en) * | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| HUE068603T2 (hu) | 2015-06-23 | 2025-01-28 | Childrens Hospital Philadelphia | Módosított IX-es faktor, valamint készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez |
| WO2017015102A1 (en) | 2015-07-17 | 2017-01-26 | The Trustees Of The University Of Pennsylvania | Compositions and methods for achieving high levels of transduction in human liver cells |
| US20190000940A1 (en) | 2015-07-31 | 2019-01-03 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of aadc deficiency |
| JP7064214B2 (ja) | 2015-09-28 | 2022-05-10 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 抗体回避性ウイルスベクターのための方法および組成物 |
| WO2017066764A2 (en) | 2015-10-16 | 2017-04-20 | William Marsh Rice University | Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses |
| WO2017075335A1 (en) | 2015-10-28 | 2017-05-04 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
| MX2018004755A (es) | 2015-10-29 | 2018-12-19 | Voyager Therapeutics Inc | Entrega de polinucleotidos dirigidos al sistema nervioso central. |
| PE20231949A1 (es) | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| RU2021102893A (ru) | 2015-11-05 | 2021-03-03 | Бамбу Терапьютикс, Инк. | Модифицированные гены атаксии фридрейха и векторы для генной терапии |
| US10406244B2 (en) | 2015-12-02 | 2019-09-10 | The Board Of Trustees Of The Leland Stanford Junior University | AAV vectors with expanded packaging capacity |
| EP3384034B1 (en) | 2015-12-02 | 2020-07-08 | The Board of Trustees of the Leland Stanford Junior University | Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism |
| DK3387137T3 (da) | 2015-12-11 | 2021-05-03 | California Inst Of Techn | Målretningspeptider til styring af adeno-associerede virusser (aav`er) |
| CN108602859B (zh) | 2015-12-14 | 2023-05-26 | 北卡罗来纳大学教堂山分校 | 增强细小病毒载体递送的修饰衣壳蛋白 |
| WO2017143100A1 (en) | 2016-02-16 | 2017-08-24 | The Board Of Trustees Of The Leland Stanford Junior University | Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies |
| WO2017161273A1 (en) | 2016-03-18 | 2017-09-21 | The Children's Hospital Of Philadelphia | Therapeutic for treatment of diseases including the central nervous system |
| US11433116B2 (en) | 2016-04-14 | 2022-09-06 | Cedars-Sinai Medical Center | GJA1 isoforms protect against metabolic stress |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| EP3448875A4 (en) | 2016-04-29 | 2020-04-08 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| AU2017267665C1 (en) | 2016-05-18 | 2023-10-05 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| EP3472196B1 (en) | 2016-06-16 | 2026-03-18 | Charité - Universitätsmedizin Berlin | Neuropeptide-expressing vectors and methods for the treatment of epilepsy |
| IL263801B2 (en) | 2016-07-26 | 2024-01-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| US11698377B2 (en) | 2016-08-15 | 2023-07-11 | Genzyme Corporation | Methods for detecting AAV |
| JP7610923B2 (ja) | 2016-09-02 | 2025-01-09 | スパーク セラピューティクス インコーポレイテッド | Ｃｎｓ障害を治療するための方法及びベクター |
| EP3293203A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| IL299325B2 (en) | 2016-09-12 | 2025-08-01 | Inst Nat Sante Rech Med | Acid-alpha glucosidase variants and uses thereof |
| EP3293259A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| US11578340B2 (en) | 2016-10-13 | 2023-02-14 | University Of Massachusetts | AAV capsid designs |
| GB2607227B (en) | 2016-10-27 | 2023-05-10 | Intima Bioscience Inc | Viral methods of T cell therapy |
| AU2018221738B2 (en) | 2017-02-15 | 2024-06-27 | The University Of North Carolina At Chapel Hill | Methods and compositions for gene transfer across the vasculature |
| US11541131B2 (en) | 2017-03-10 | 2023-01-03 | Genethon | Treatment of glycogen storage disease III |
| ES2966692T3 (es) | 2017-03-15 | 2024-04-23 | Univ North Carolina Chapel Hill | Vectores de virus adenoasociados poliploides y métodos de fabricación y uso de los mismos |
| WO2018189244A1 (en) | 2017-04-11 | 2018-10-18 | Ruprecht-Karls-Universität Heidelberg | Adeno-associated virus library |
| WO2018200419A1 (en) | 2017-04-23 | 2018-11-01 | The Trustees Of The University Of Pennsylvania | Viral vectors comprising engineered aav capsids and compositions containing the same |
| CA3061652A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CN111108198A (zh) | 2017-05-05 | 2020-05-05 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
| AU2018281306B2 (en) | 2017-06-07 | 2025-04-17 | Spark Therapeutics, Inc. | Enhancing agents for improved cell transfection and/or rAAV vector production |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| MX2020000216A (es) | 2017-06-30 | 2020-09-03 | Spark Therapeutics Inc | Metodos de purificacion por columna de vectores aav. |
| TWI820034B (zh) | 2017-07-31 | 2023-11-01 | 香港商映像生物有限公司 | 眼部疾病之細胞模式及用於眼部疾病的療法 |
| BR112020001979A2 (pt) | 2017-08-01 | 2020-08-18 | Spark Therapeutics, Inc. | métodos de terapia genética do fator viii (fviii) |
| WO2019028306A2 (en) | 2017-08-03 | 2019-02-07 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES |
| CA3070040A1 (en) | 2017-08-29 | 2019-03-07 | Ruprecht-Karls-Universitat Heidelberg | Rationally designed virus-like particles for modulation of chimeric antigen receptor (car)-t-cell therapy |
| US20200377887A1 (en) | 2017-09-22 | 2020-12-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| EP3694998A1 (en) | 2017-10-12 | 2020-08-19 | Freeline Therapeutics Limited | Life-cycle-defective adenovirus helper viruses, their production and use for producing raav |
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