HUE042693T2 - Kapszid - Google Patents
KapszidInfo
- Publication number
- HUE042693T2 HUE042693T2 HUE16721917A HUE16721917A HUE042693T2 HU E042693 T2 HUE042693 T2 HU E042693T2 HU E16721917 A HUE16721917 A HU E16721917A HU E16721917 A HUE16721917 A HU E16721917A HU E042693 T2 HUE042693 T2 HU E042693T2
- Authority
- HU
- Hungary
- Prior art keywords
- capsid
- Prior art date
Links
- 210000000234 capsid Anatomy 0.000 title 1
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)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biophysics (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Virology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (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 (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
GBGB1508026.0A GB201508026D0 (en) | 2015-05-11 | 2015-05-11 | Capsid |
Publications (1)
Publication Number | Publication Date |
---|---|
HUE042693T2 true HUE042693T2 (hu) | 2019-07-29 |
Family
ID=53489438
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HUE16721917A HUE042693T2 (hu) | 2015-05-11 | 2016-05-10 | Kapszid |
Country Status (18)
Country | Link |
---|---|
US (3) | US10414803B2 (hu) |
EP (2) | EP3511021A1 (hu) |
JP (2) | JP6619454B2 (hu) |
KR (1) | KR20180019543A (hu) |
CN (1) | CN107864653B (hu) |
AU (1) | AU2016261454B2 (hu) |
CA (1) | CA2985945C (hu) |
DK (1) | DK3250239T3 (hu) |
ES (1) | ES2711349T3 (hu) |
GB (1) | GB201508026D0 (hu) |
HR (1) | HRP20190442T1 (hu) |
HU (1) | HUE042693T2 (hu) |
LT (1) | LT3250239T (hu) |
PL (1) | PL3250239T3 (hu) |
PT (1) | PT3250239T (hu) |
TR (1) | TR201901582T4 (hu) |
TW (1) | TW201704253A (hu) |
WO (1) | WO2016181123A1 (hu) |
Families Citing this family (43)
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 |
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 |
SG11202009450SA (en) | 2018-04-03 | 2020-10-29 | Stridebio Inc | Virus vectors for targeting ophthalmic tissues |
BR112020020195A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
KR20210006357A (ko) | 2018-04-03 | 2021-01-18 | 스트라이드바이오 인코포레이티드 | 항체-회피 바이러스 벡터 |
US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | 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 |
EP4269579A3 (en) | 2019-01-04 | 2024-01-24 | Ultragenyx Pharmaceutical Inc. | Gene therapy constructs for treating wilson disease |
MX2021011468A (es) | 2019-03-21 | 2021-12-15 | Vectores de virus adenoasociados recombinantes. | |
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 |
US20220162642A1 (en) | 2019-04-12 | 2022-05-26 | Freeline Therapeutics Limited | Plasmid system |
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 |
EP4022073A4 (en) * | 2019-08-29 | 2023-12-20 | The Board Of Trustees Of The Leland Stanford Junior University | ADENO-ASSOCIATED VIRAL VECTORS TO CROSS THE HUMAN BLOOD-BRAIN BARRIER |
US11905523B2 (en) | 2019-10-17 | 2024-02-20 | Ginkgo Bioworks, Inc. | Adeno-associated viral vectors for treatment of Niemann-Pick Disease type-C |
US20230093183A1 (en) * | 2020-02-25 | 2023-03-23 | Children's Medical Research Institute | Adeno-associated virus capsid polypeptides and vectors |
CN116209768A (zh) * | 2020-07-03 | 2023-06-02 | 吉尼松公司 | 用于通过高变区交换工程化新杂合aav衣壳的方法 |
CN111876432B (zh) * | 2020-07-29 | 2022-10-28 | 舒泰神(北京)生物制药股份有限公司 | 一组肝靶向新型腺相关病毒的获得及其应用 |
CN116745409A (zh) | 2020-08-19 | 2023-09-12 | 萨雷普塔治疗公司 | 用于治疗雷特综合征的腺相关病毒载体 |
EP4213890A1 (en) | 2020-09-15 | 2023-07-26 | RegenxBio Inc. | Vectorized lanadelumab and administration thereof |
WO2022060916A1 (en) | 2020-09-15 | 2022-03-24 | 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 |
CN116528892A (zh) | 2020-10-28 | 2023-08-01 | 再生生物股份有限公司 | 用于眼部适应症的载体化抗TNF-α抗体 |
US20230390418A1 (en) | 2020-10-29 | 2023-12-07 | 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 |
WO2023072181A1 (en) * | 2021-10-28 | 2023-05-04 | Shanghai Vitalgen Biopharma Co., Ltd. | Adeno-associated virus capsid |
GB202205514D0 (en) | 2022-04-13 | 2022-05-25 | Freeline Therapeutics Ltd | Mechanical lysis |
WO2023215807A1 (en) | 2022-05-03 | 2023-11-09 | Regenxbio Inc. | VECTORIZED ANTI-TNF-α INHIBITORS FOR OCULAR INDICATIONS |
AR129215A1 (es) | 2022-05-03 | 2024-07-31 | Regenxbio Inc | Anticuerpos anti-complemento y agentes del complemento vectorizados y administración de estos |
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 |
WO2024238867A1 (en) | 2023-05-16 | 2024-11-21 | 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 |
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 |
AU1449499A (en) | 1997-10-31 | 1999-05-24 | Maxygen, Incorporated | Modification of virus tropism and host range by viral genome shuffling |
DE69941905D1 (de) | 1998-11-10 | 2010-02-25 | 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 |
EP1048726B1 (en) | 1999-04-27 | 2006-07-26 | Négrier, Claude | Modified factor VIII cDNA |
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 |
JP2003526377A (ja) | 2000-03-14 | 2003-09-09 | ニユーロロジクス・インコーポレーテツド | キメラキャプシドベクターの製造 |
US7749492B2 (en) | 2001-01-05 | 2010-07-06 | Nationwide Children's Hospital, Inc. | AAV vectors and methods |
US6962815B2 (en) | 2001-01-05 | 2005-11-08 | Children's Hopital Inc. | AAV2 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 |
MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
EP1453547B1 (en) | 2001-12-17 | 2016-09-21 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
AU2003223775A1 (en) | 2002-04-30 | 2003-11-17 | Duke University | Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use |
DK2357189T3 (en) | 2003-06-19 | 2017-06-19 | Genzyme Corp | AAV virions with reduced immunoreactivity and applications thereof |
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 |
CA2537793C (en) | 2003-09-30 | 2013-07-23 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
US20060166363A1 (en) | 2004-01-27 | 2006-07-27 | Sergei Zolotukhin | Modified baculovirus expression system for production of pseudotyped rAAV vector |
CN101124328A (zh) | 2004-12-15 | 2008-02-13 | 北卡罗来纳查佩尔山大学 | 嵌合载体 |
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 |
WO2007120542A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid library and aav capsid proteins |
CA2660963A1 (en) | 2006-08-21 | 2008-02-28 | Genentech, Inc. | Aza-benzothiophenyl compounds and methods of use |
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 |
HRP20161150T1 (hr) | 2007-04-09 | 2016-11-18 | University Of Florida Research Foundation, Inc. | PRIPRAVCI rAAV VEKTORA KOJI IMAJU TIROZIN-MODIFICIRANE CAPSID PROTEINE I POSTUPCI ZA UPORABU |
DK2158211T3 (en) * | 2007-05-31 | 2016-12-05 | Medigene Ag | Mutated structural protein of a parvovirus |
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 |
ES2634118T3 (es) | 2009-02-11 | 2017-09-26 | The University Of North Carolina At Chapel Hill | Vectores de virus modificados y métodos para fabricar y utilizar los mismos |
PL2403867T3 (pl) | 2009-03-04 | 2019-12-31 | Deutsches Krebsforschungszentrum | Białko aktywujące składanie (aap) i jego zastosowanie do wytwarzania cząstek parwowirusa zasadniczo składających się z vp3 |
GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
EP3147295B2 (en) | 2011-08-24 | 2023-11-22 | The Board of Trustees of the Leland Stanford Junior University | New avv capsid proteins for nucleic acid transfer |
WO2013063379A1 (en) | 2011-10-28 | 2013-05-02 | University Of North Carolina At Chapel Hill | Cell line for production of adeno-associated virus |
EP3738974A1 (en) | 2012-09-28 | 2020-11-18 | The University of North Carolina at Chapel Hill | Aav vectors targeted to oligodendrocytes |
WO2014064277A1 (en) | 2012-10-26 | 2014-05-01 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
ES2667209T3 (es) | 2013-03-14 | 2018-05-10 | Asklepios Biopharmaceutical, Inc. | Genes del receptor-1 de VEGF soluble modificado y vectores para terapia génica |
PT2968605T (pt) | 2013-03-15 | 2022-09-22 | Univ North Carolina Chapel Hill | Métodos e composições para vetores aav de ligação dupla de glicano |
CA2917018A1 (en) | 2013-05-21 | 2014-12-04 | University Of Florida Research Foundation, Inc. | Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer |
KR20220041963A (ko) | 2013-07-22 | 2022-04-01 | 더 칠드런스 호스피탈 오브 필라델피아 | 변종 aav 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도 |
HUE061629T2 (hu) | 2013-08-27 | 2023-07-28 | Res Inst Nationwide Childrens Hospital | Termékek és módszerek amyotrophiás lateralis sclerosis kezelésére |
NZ718926A (en) * | 2013-10-11 | 2021-12-24 | Massachusetts Eye & Ear Infirmary | Methods of predicting ancestral virus sequences and uses thereof |
GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
US20170007720A1 (en) | 2014-02-21 | 2017-01-12 | University Of Florida Research Foundation, Inc. | Methods and compositions for gene delivery to on bipolar cells |
CA2941640A1 (en) | 2014-03-04 | 2015-09-11 | University Of Florida Research Foundation, Inc. | Improved raav vectors and methods for transduction of photoreceptors and rpe cells |
EP3134530B1 (en) | 2014-04-25 | 2019-06-12 | Genethon | Treatment of hyperbilirubinemia |
AU2015252797C1 (en) | 2014-05-02 | 2021-04-22 | Genzyme Corporation | AAV vectors for retinal and CNS gene therapy |
WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
CN107073051B (zh) | 2014-10-21 | 2021-08-24 | 马萨诸塞大学 | 重组aav变体及其用途 |
GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
EP3221453A4 (en) | 2014-11-21 | 2018-09-12 | The University of North Carolina at Chapel Hill | Aav vectors targeted to the central nervous system |
GB201502137D0 (en) | 2015-02-09 | 2015-03-25 | Ucl Business Plc | Treatment |
US10648000B2 (en) | 2015-02-16 | 2020-05-12 | University Of Florida Research Foundation, Incorporated | rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes |
EP3262162A4 (en) | 2015-02-23 | 2018-08-08 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
BR112017018846A2 (pt) | 2015-03-02 | 2018-07-31 | Adverum Biotechnologies, Inc. | composições e métodos para entrega intravítrea de polinucleotídeos a cones retinianos. |
MX388331B (es) | 2015-03-24 | 2025-03-19 | Univ California | Variantes de virus adenoasociado y métodos de uso de estas |
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 |
CA3019315A1 (en) | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
GB201508026D0 (en) * | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
JP6831779B2 (ja) | 2015-06-23 | 2021-02-17 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia | 修飾された第ix因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用 |
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) |
HK1258413A1 (zh) | 2015-10-29 | 2019-11-08 | Voyager Therapeutics, Inc. | 遞送中樞神經系統(cns)靶向多核苷酸 |
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 |
CN108348621A (zh) | 2015-11-05 | 2018-07-31 | 竹治疗股份有限公司 | 用于基因治疗的经修饰的弗里德赖希共济失调基因及载体 |
US10406244B2 (en) | 2015-12-02 | 2019-09-10 | The Board Of Trustees Of The Leland Stanford Junior University | AAV vectors with expanded packaging capacity |
WO2017096164A1 (en) | 2015-12-02 | 2017-06-08 | The Board Of Trustees Of The Leland Stanford Junior University | Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism |
JP7066619B2 (ja) | 2015-12-11 | 2022-05-13 | カリフォルニア インスティチュート オブ テクノロジー | アデノ随伴ウイルス(aav)を誘導するための標的指向性ペプチド |
RU2751953C2 (ru) | 2015-12-14 | 2021-07-21 | Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл | Модифицированные капсидные белки для улучшения доставки парвовирусных векторов |
AU2017219865B2 (en) | 2016-02-16 | 2023-04-13 | The Board Of Trustees Of The Leland Stanford Junior University | Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies |
EP3429605A4 (en) | 2016-03-18 | 2019-09-18 | The Children's Hospital of Philadelphia | THERAPEUTIC FOR THE TREATMENT OF DISEASES INCLUDING THE CENTRAL NERVOUS SYSTEM |
WO2017180896A1 (en) | 2016-04-14 | 2017-10-19 | Cedars-Sinai Medical Center | Gja1 isoforms protect against metabolic stress |
US20190224339A1 (en) | 2016-04-29 | 2019-07-25 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
WO2017189959A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
CA3024448A1 (en) | 2016-05-18 | 2017-11-23 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
CN109843913B (zh) | 2016-06-16 | 2024-03-08 | 柏林夏洛特医科大学 | 神经肽表达载体以及用于治疗癫痫的方法 |
EP3491008A2 (en) | 2016-07-26 | 2019-06-05 | BioMarin Pharmaceutical Inc. | Novel adeno-associated virus capsid proteins |
DK3497207T3 (da) | 2016-08-15 | 2021-03-22 | Genzyme Corp | Fremgangsmåder til påvisning af aav |
MX2019002518A (es) | 2016-09-02 | 2019-07-18 | Spark Therapeutics Inc | Metodos y vectores para el tratamiento de trastornos del snc. |
KR20240001721A (ko) | 2016-09-12 | 2024-01-03 | 제네똥 | 산-알파 글루코시다제 변이체 및 이의 용도 |
EP3293203A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
EP3293259A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
WO2018071831A1 (en) | 2016-10-13 | 2018-04-19 | University Of Massachusetts | Aav capsid designs |
AU2017347854B2 (en) | 2016-10-27 | 2022-12-08 | Intima Bioscience, Inc. | Viral methods of T cell therapy |
EA038695B1 (ru) | 2017-02-15 | 2021-10-06 | Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл | Способы и композиции для переноса генов по сосудистой сети |
US11541131B2 (en) | 2017-03-10 | 2023-01-03 | Genethon | Treatment of glycogen storage disease III |
AU2018234695B2 (en) | 2017-03-15 | 2024-10-03 | The University Of North Carolina At Chapel Hill | Polyploid adeno-associated virus vectors and methods of making and using the same |
US20200370039A1 (en) | 2017-04-11 | 2020-11-26 | Ruprecht-Karls-Universitat 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 |
JP2020518258A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. | 筋萎縮性側索硬化症(als)治療組成物および方法 |
US11752181B2 (en) | 2017-05-05 | 2023-09-12 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
EP3635108A4 (en) | 2017-06-07 | 2021-03-31 | Spark Therapeutics, Inc. | ACTIVATION AGENTS INTENDED FOR ENHANCED CELL TRANSFECTION AND / OR RAAV VECTOR PRODUCTION |
JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
CA3068622A1 (en) | 2017-06-30 | 2019-01-03 | Spark Therapeutics, Inc. | Aav vector column purification methods |
SG11202000840YA (en) | 2017-07-31 | 2020-02-27 | Reflection Biotechnologies Ltd | Cellular models of and therapies for ocular diseases |
WO2019028192A1 (en) | 2017-08-01 | 2019-02-07 | Spark Therapeutics, Inc. | METHODS OF GENE THERAPY TARGETING FACTOR VIII (FVIII) |
TWI722310B (zh) | 2017-08-03 | 2021-03-21 | 美商航海家醫療公司 | 用於aav之遞送之組合物及方法 |
CN111132993A (zh) | 2017-08-29 | 2020-05-08 | 海德堡鲁普雷希特卡尔斯大学 | 合理设计的用于调节嵌合抗原受体(car)-t细胞疗法的病毒样颗粒 |
EP3684388A4 (en) | 2017-09-22 | 2021-09-22 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT OF HUNTINGTON'S DISEASE |
WO2019073059A1 (en) | 2017-10-12 | 2019-04-18 | Freeline Therapeutics Limited | ADENOVIRAL AUXILIARY VIRUSES WITH DEFECTIVE CYCLE LIFE, PRODUCTION AND USE FOR THE PRODUCTION OF RAAV |
-
2015
- 2015-05-11 GB GBGB1508026.0A patent/GB201508026D0/en not_active Ceased
-
2016
- 2016-05-10 HU HUE16721917A patent/HUE042693T2/hu unknown
- 2016-05-10 US US15/573,350 patent/US10414803B2/en active Active
- 2016-05-10 WO PCT/GB2016/051329 patent/WO2016181123A1/en active Application Filing
- 2016-05-10 TR TR2019/01582T patent/TR201901582T4/tr unknown
- 2016-05-10 LT LTEP16721917.9T patent/LT3250239T/lt unknown
- 2016-05-10 KR KR1020177035515A patent/KR20180019543A/ko not_active Ceased
- 2016-05-10 CA CA2985945A patent/CA2985945C/en active Active
- 2016-05-10 ES ES16721917T patent/ES2711349T3/es active Active
- 2016-05-10 EP EP18210522.1A patent/EP3511021A1/en active Pending
- 2016-05-10 HR HRP20190442TT patent/HRP20190442T1/hr unknown
- 2016-05-10 AU AU2016261454A patent/AU2016261454B2/en active Active
- 2016-05-10 CN CN201680027100.0A patent/CN107864653B/zh active Active
- 2016-05-10 PL PL16721917T patent/PL3250239T3/pl unknown
- 2016-05-10 EP EP16721917.9A patent/EP3250239B1/en active Active
- 2016-05-10 PT PT16721917T patent/PT3250239T/pt unknown
- 2016-05-10 JP JP2017558723A patent/JP6619454B2/ja active Active
- 2016-05-10 DK DK16721917.9T patent/DK3250239T3/en active
- 2016-05-11 TW TW105114580A patent/TW201704253A/zh unknown
-
2019
- 2019-08-15 US US16/541,516 patent/US12138318B2/en active Active
- 2019-11-14 JP JP2019206191A patent/JP2020096587A/ja active Pending
-
2024
- 2024-10-09 US US18/911,006 patent/US20250127922A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
DK3250239T3 (en) | 2019-03-11 |
US12138318B2 (en) | 2024-11-12 |
WO2016181123A1 (en) | 2016-11-17 |
JP2020096587A (ja) | 2020-06-25 |
EP3250239A1 (en) | 2017-12-06 |
US10414803B2 (en) | 2019-09-17 |
US20200079821A1 (en) | 2020-03-12 |
KR20180019543A (ko) | 2018-02-26 |
AU2016261454A1 (en) | 2017-11-23 |
CN107864653B (zh) | 2022-06-21 |
CA2985945A1 (en) | 2016-11-17 |
US20180111965A1 (en) | 2018-04-26 |
CA2985945C (en) | 2022-09-06 |
LT3250239T (lt) | 2019-03-25 |
PT3250239T (pt) | 2019-02-19 |
PL3250239T3 (pl) | 2019-05-31 |
TW201704253A (zh) | 2017-02-01 |
US20250127922A1 (en) | 2025-04-24 |
EP3250239B1 (en) | 2018-12-12 |
GB201508026D0 (en) | 2015-06-24 |
JP2018520997A (ja) | 2018-08-02 |
HRP20190442T1 (hr) | 2019-05-03 |
CN107864653A (zh) | 2018-03-30 |
JP6619454B2 (ja) | 2019-12-11 |
ES2711349T3 (es) | 2019-05-03 |
TR201901582T4 (tr) | 2019-02-21 |
EP3511021A1 (en) | 2019-07-17 |
AU2016261454B2 (en) | 2019-07-11 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
HUE042693T2 (hu) | Kapszid | |
DK3283625T3 (da) | Nukleasemedieret genomeditering | |
DK3331869T3 (da) | Muscarinagonister | |
DK3277719T3 (da) | Polypeptider | |
EP3316375A4 (en) | Redox flow cell | |
DK3331528T3 (da) | Muskarinagonister | |
DK3305788T3 (da) | Janus-kinase-hæmmer | |
DK3360890T3 (da) | Genterapi | |
DK3331529T3 (da) | Muskarinagonister | |
DK3310450T3 (da) | Olie-vand-separator | |
EP3290364A4 (en) | Conveyor belt wear monitoring system | |
DK3270930T3 (da) | Præeklampsi | |
EP3381654A4 (en) | Liquid blow molding method | |
DK3394281T3 (da) | Gærcelle | |
EP3363322A4 (en) | Hair extender | |
DK3292136T3 (da) | Penicillin-g-acylaser | |
DK3274482T3 (da) | Stenborsknop | |
DE112015006486A5 (de) | Inkubationsrinne | |
EP3377179A4 (en) | DEUTERED EPI-743 | |
EP3548009A4 (en) | POLYMERIC NANOPARTICLES | |
DK3318308T3 (da) | Badmintonketsjer | |
DE112015005905A5 (de) | Gurtschlossbringer | |
DE112016005599A5 (de) | Strahlungsgrill | |
ES1140083Y (es) | Descompatactador tetrafunción | |
DE102015206660A8 (de) | Plattenaufteilanlage |