PT3250239T - Cápside - Google Patents

Cápside

Info

Publication number
PT3250239T
PT3250239T PT16721917T PT16721917T PT3250239T PT 3250239 T PT3250239 T PT 3250239T PT 16721917 T PT16721917 T PT 16721917T PT 16721917 T PT16721917 T PT 16721917T PT 3250239 T PT3250239 T PT 3250239T
Authority
PT
Portugal
Prior art keywords
capsid
Prior art date
Application number
PT16721917T
Other languages
English (en)
Portuguese (pt)
Inventor
Nathwani Amit
Dane Allison
Original Assignee
Ucl Business Plc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Ucl Business Plc filed Critical Ucl Business Plc
Publication of PT3250239T publication Critical patent/PT3250239T/pt

Links

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)
PT16721917T 2015-05-11 2016-05-10 Cápside PT3250239T (pt)

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
PT3250239T true PT3250239T (pt) 2019-02-19

Family

ID=53489438

Family Applications (1)

Application Number Title Priority Date Filing Date
PT16721917T PT3250239T (pt) 2015-05-11 2016-05-10 Cápside

Country Status (18)

Country Link
US (3) US10414803B2 (enExample)
EP (2) EP3511021A1 (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)

* Cited by examiner, † Cited by third party
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
CN114606267A (zh) 2015-09-28 2022-06-10 北卡罗来纳-查佩尔山大学 逃避抗体的病毒载体的方法和组合物
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
EP4361277A3 (en) 2016-12-30 2024-07-31 The Trustees of The University of Pennsylvania Gene therapy for treating wilson's disease
WO2019006418A2 (en) * 2017-06-30 2019-01-03 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
CN112004820B (zh) * 2018-03-30 2023-09-12 利兰斯坦福初级大学董事会 具有增强的人胰腺向性的新型重组腺相关病毒衣壳
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
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
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
DK3906066T5 (da) 2019-01-04 2024-08-05 Ultragenyx Pharmaceutical Inc Geneterapikonstruktioner til behandling af wilsons sygdom
EA202192501A1 (ru) 2019-03-21 2021-12-16 Страйдбайо, Инк. Рекомбинантные аденоассоциированные вирусные векторы
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
EP3722434B1 (en) 2019-04-12 2022-07-27 Freeline Therapeutics Limited Plasmid system
JP7562558B2 (ja) 2019-04-12 2024-10-07 アセンド アドヴァンスト セラピーズ リミテッド プラスミドシステム
JP7371954B2 (ja) * 2019-07-12 2023-10-31 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
AU2020330121B2 (en) * 2019-08-14 2025-10-30 University Of Florida Research Foundation, Incorporated AAV capsid variants for gene therapy
CN115087738A (zh) * 2019-08-29 2022-09-20 小利兰·斯坦福大学托管委员会 用于穿越人血脑屏障的腺相关病毒载体
BR112022006530A2 (pt) 2019-10-17 2022-07-05 Stridebio Inc Vetores virais adenoassociados para o tratamento da doença de niemann-pick tipo c
US20230093183A1 (en) * 2020-02-25 2023-03-23 Children's Medical Research Institute Adeno-associated virus capsid polypeptides and vectors
JP2023532704A (ja) * 2020-07-03 2023-07-31 ジェネトン 超可変領域スワッピングを通じて新規のハイブリッドａａｖカプシドを操作する方法
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
TW202227632A (zh) 2020-08-19 2022-07-16 美商史崔德生物公司 用於治療雷特症候群之腺相關病毒載體
EP4213890A1 (en) 2020-09-15 2023-07-26 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
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
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
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
EP4518972A2 (en) 2022-05-03 2025-03-12 RegenxBio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
EP4518973A1 (en) 2022-05-03 2025-03-12 RegenxBio Inc. Vectorized anti-tnf-alpha inhibitors for ocular indications
GB202214328D0 (en) 2022-09-29 2022-11-16 Freeline Therapeutics Ltd Method
WO2024238867A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents 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
WO2025217230A1 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
WO2025238067A1 (en) 2024-05-15 2025-11-20 Ascend Advanced Therapies Gmbh Novel vector system for aav production

Family Cites Families (101)

* Cited by examiner, † Cited by third party
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
US6596539B1 (en) 1997-10-31 2003-07-22 Maxygen, Inc. Modification of virus tropism and host range by viral genome shuffling
WO2000028004A1 (en) 1998-11-10 2000-05-18 The University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
EP1010762A1 (en) 1998-12-02 2000-06-21 Aventis Behring Gesellschaft mit beschränkter Haftung DNA constructs of blood clotting factors and P-Selectin
EP1026250A1 (en) 1998-12-02 2000-08-09 Aventis Behring GmbH 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
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
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
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
WO2003092594A2 (en) 2002-04-30 2003-11-13 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
HUE054046T2 (hu) 2003-06-19 2021-08-30 Genzyme Corp Csökkentett immunreaktivitású AAV virionok és alkalmazásaik
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
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
JP5136766B2 (ja) 2004-12-15 2013-02-06 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル キメラベクター
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
JP5349306B2 (ja) 2006-08-21 2013-11-20 ジェネンテック， インコーポレイテッド アザベンゾチオフェニル化合物および使用方法
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
WO2008124724A1 (en) 2007-04-09 2008-10-16 University Of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
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
CN102341406B (zh) 2009-03-04 2016-06-08 德国癌症研究中心 组装活化蛋白（aap）及其用于制备基本上由vp3组成的细小病毒颗粒的应用
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
EP2771455B1 (en) 2011-10-28 2016-10-05 The University of North Carolina At Chapel Hill Cell line for production of adeno-associated virus
EP2900686B1 (en) 2012-09-28 2020-06-10 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
IL293294B2 (en) 2013-03-15 2023-04-01 Univ North Carolina Chapel Hill Methods and compounds for ligation of paired aav glycan vectors
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
PL3024498T3 (pl) 2013-07-22 2020-05-18 The Children's Hospital Of Philadelphia Wariant aav oraz kompozycje, sposoby i zastosowania transferu genów do komórek, narządów i tkanek
ES2936220T3 (es) 2013-08-27 2023-03-15 Res Inst Nationwide Childrens Hospital Productos y métodos para el tratamiento de la esclerosis lateral amiotrófica
HUE052676T2 (hu) * 2013-10-11 2021-05-28 Massachusetts Eye & Ear Infirmary Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik
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
EP3113787B1 (en) 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
CA2942451A1 (en) 2014-04-25 2015-10-29 Genethon Treatment of hyperbilirubinemia
CN114231563B (zh) 2014-05-02 2024-08-16 建新公司 用于视网膜及cns基因疗法的aav载体
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
RU2020140209A (ru) 2014-10-21 2021-01-25 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
CN114480440A (zh) 2014-11-21 2022-05-13 北卡罗来纳-查佩尔山大学 靶向中枢神经系统的aav载体
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
WO2016137949A1 (en) 2015-02-23 2016-09-01 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
US11021519B2 (en) 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
CN107532177A (zh) 2015-03-24 2018-01-02 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
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
EP3285788B1 (en) 2015-04-23 2024-12-18 University of Massachusetts Modulation of aav vector transgene expression
GB201508026D0 (en) * 2015-05-11 2015-06-24 Ucl Business Plc Capsid
US10799566B2 (en) 2015-06-23 2020-10-13 The Children's Hospital Of Philadelphia Modified factor IX, and compositions, methods and uses for gene transfer to cells, organs, and tissues
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
WO2017023724A1 (en) 2015-07-31 2017-02-09 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
CN114606267A (zh) 2015-09-28 2022-06-10 北卡罗来纳-查佩尔山大学 逃避抗体的病毒载体的方法和组合物
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
US20180230489A1 (en) 2015-10-28 2018-08-16 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
EP3370778A1 (en) 2015-11-05 2018-09-12 Bamboo Therapeutics, Inc. Modified friedreich ataxia genes and vectors for gene therapy
US10385320B2 (en) 2015-12-02 2019-08-20 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
CN108699565B (zh) 2015-12-11 2023-08-08 加州理工学院 用于定向腺相关病毒(aav)的靶向肽
WO2017106236A1 (en) 2015-12-14 2017-06-22 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
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
US11155817B2 (en) 2016-03-18 2021-10-26 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
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448875A4 (en) 2016-04-29 2020-04-08 Voyager Therapeutics, Inc. Compositions for the treatment of disease
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2017216301A1 (en) 2016-06-16 2017-12-21 Charité Berlin Neuropeptide-expressing vectors and methods for the treatment of epilepsy
BR112019001532A2 (pt) 2016-07-26 2019-09-10 Biomarin Pharm Inc proteínas de capsídeo de vírus adenoassociado inovadoras
TN2019000047A1 (en) 2016-08-15 2020-07-15 Genzyme Corp Methods for detecting aav
BR112019004353A2 (pt) 2016-09-02 2019-05-28 Spark Therapeutics Inc métodos e vetores para tratar distúrbios do cns
EP3293203A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
PE20191203A1 (es) 2016-09-12 2019-09-10 Genethon Variantes de alfa-glucosidasa acida y usos de las mismas
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
JP2019531755A (ja) 2016-10-27 2019-11-07 インティマ バイオサイエンス， インコーポレイテッド 遺伝子改変細胞を作製するウイルス法
JP7291397B2 (ja) 2017-02-15 2023-06-15 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル 血管系を通過する遺伝子移入の方法および組成物
MA47820A (fr) 2017-03-10 2020-01-15 Genethon Traitement de la glycogénose de type iii
CN117801075A (zh) 2017-03-15 2024-04-02 北卡罗来纳-查佩尔山大学 多倍体腺相关病毒载体及其制备和使用方法
EP3609542B1 (en) 2017-04-11 2023-02-22 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
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US12404526B2 (en) 2017-06-07 2025-09-02 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
WO2019006390A1 (en) 2017-06-30 2019-01-03 Spark Therapeutics, Inc. AAV VECTOR COLUMN PURIFICATION METHOD
US20200255859A1 (en) 2017-07-31 2020-08-13 Reflection Biotechnologies Limited Cellular models of and therapies for ocular diseases
SG11202000650YA (en) 2017-08-01 2020-02-27 Spark Therapeutics Inc Factor viii (fviii) gene therapy methods
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
EP3684388A4 (en) 2017-09-22 2021-09-22 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF HUNTINGTON'S DISEASE
US12123027B2 (en) 2017-10-12 2024-10-22 Ascend Advanced Therapies Limited Life-cycle-defective adenovirus helper viruses, their production and use for producing rAAV

Also Published As

Publication number Publication date
TW201704253A (zh) 2017-02-01
EP3250239B1 (en) 2018-12-12
US20250127922A1 (en) 2025-04-24
US10414803B2 (en) 2019-09-17
LT3250239T (lt) 2019-03-25
PL3250239T3 (pl) 2019-05-31
EP3250239A1 (en) 2017-12-06
JP6619454B2 (ja) 2019-12-11
CN107864653A (zh) 2018-03-30
CA2985945A1 (en) 2016-11-17
WO2016181123A1 (en) 2016-11-17
TR201901582T4 (tr) 2019-02-21
AU2016261454B2 (en) 2019-07-11
CN107864653B (zh) 2022-06-21
ES2711349T3 (es) 2019-05-03
JP2020096587A (ja) 2020-06-25
EP3511021A1 (en) 2019-07-17
AU2016261454A1 (en) 2017-11-23
HUE042693T2 (hu) 2019-07-29
KR20180019543A (ko) 2018-02-26
GB201508026D0 (en) 2015-06-24
US20180111965A1 (en) 2018-04-26
JP2018520997A (ja) 2018-08-02
DK3250239T3 (en) 2019-03-11
US20200079821A1 (en) 2020-03-12
CA2985945C (en) 2022-09-06
HRP20190442T1 (hr) 2019-05-03
US12138318B2 (en) 2024-11-12

Similar Documents

Publication Publication Date Title
PL3250239T3 (pl) Kapsyd
GB201506824D0 (en) TrailReach Multitrial
GB201406608D0 (en) Virus
GB201511039D0 (en) In-vitro-analyser
GB201419572D0 (en) Virus
GB201513637D0 (en) Pooppot
GB201515850D0 (en) Glycommetics
GB201502617D0 (en) Supports
GB201620212D0 (en) Gargeraet
GB201519328D0 (en) Drydam
GB201518442D0 (en) Drumstring
GB201517942D0 (en) Projectacase
GB201517489D0 (en) NoFlat
GB201516026D0 (en) Rencu
GB201516060D0 (en) Microsecurity
GB201515187D0 (en) Kerbie
GB201514277D0 (en) Tippex
GB201514156D0 (en) Elicrofft
GB201507029D0 (en) Supports
GB201513697D0 (en) Megablaster
GB201513704D0 (en) Hardwater
GB201513721D0 (en) Srays
GB201513227D0 (en) Flaskettle
GB201513199D0 (en) Ezipost
GB201511779D0 (en) G-track