CA2985945C - Capsid - Google Patents

Capsid Download PDF

Info

Publication number
CA2985945C
CA2985945C CA2985945A CA2985945A CA2985945C CA 2985945 C CA2985945 C CA 2985945C CA 2985945 A CA2985945 A CA 2985945A CA 2985945 A CA2985945 A CA 2985945A CA 2985945 C CA2985945 C CA 2985945C
Authority
CA
Canada
Prior art keywords
aav
sequence
disease
capsid protein
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.)
Active
Application number
CA2985945A
Other languages
English (en)
French (fr)
Other versions
CA2985945A1 (en
Inventor
Amit Nathwani
Allison DANE
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
UCL Business Ltd
Original Assignee
UCL Business Ltd
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 Ltd filed Critical UCL Business Ltd
Publication of CA2985945A1 publication Critical patent/CA2985945A1/en
Application granted granted Critical
Publication of CA2985945C publication Critical patent/CA2985945C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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)
CA2985945A 2015-05-11 2016-05-10 Capsid Active CA2985945C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1508026.0 2015-05-11
GBGB1508026.0A GB201508026D0 (en) 2015-05-11 2015-05-11 Capsid
PCT/GB2016/051329 WO2016181123A1 (en) 2015-05-11 2016-05-10 Capsid

Publications (2)

Publication Number Publication Date
CA2985945A1 CA2985945A1 (en) 2016-11-17
CA2985945C true CA2985945C (en) 2022-09-06

Family

ID=53489438

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2985945A Active CA2985945C (en) 2015-05-11 2016-05-10 Capsid

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)

* 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
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)

* 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
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

Also Published As

Publication number Publication date
KR20180019543A (ko) 2018-02-26
LT3250239T (lt) 2019-03-25
PT3250239T (pt) 2019-02-19
EP3250239B1 (en) 2018-12-12
TR201901582T4 (tr) 2019-02-21
HUE042693T2 (hu) 2019-07-29
GB201508026D0 (en) 2015-06-24
EP3511021A1 (en) 2019-07-17
AU2016261454B2 (en) 2019-07-11
US20180111965A1 (en) 2018-04-26
CA2985945A1 (en) 2016-11-17
US12138318B2 (en) 2024-11-12
US20200079821A1 (en) 2020-03-12
JP6619454B2 (ja) 2019-12-11
HRP20190442T1 (hr) 2019-05-03
JP2020096587A (ja) 2020-06-25
WO2016181123A1 (en) 2016-11-17
US20250127922A1 (en) 2025-04-24
ES2711349T3 (es) 2019-05-03
AU2016261454A1 (en) 2017-11-23
US10414803B2 (en) 2019-09-17
PL3250239T3 (pl) 2019-05-31
CN107864653B (zh) 2022-06-21
TW201704253A (zh) 2017-02-01
CN107864653A (zh) 2018-03-30
DK3250239T3 (en) 2019-03-11
EP3250239A1 (en) 2017-12-06
JP2018520997A (ja) 2018-08-02

Similar Documents

Publication Publication Date Title
US20250127922A1 (en) Capsid
US20220331409A1 (en) Factor ix gene therapy
Balakrishnan et al. Basic biology of adeno-associated virus (AAV) vectors used in gene therapy
Grieger et al. Adeno-associated virus as a gene therapy vector: vector development, production and clinical applications
JP5305913B2 (ja) 昆虫細胞で産生される改良されたａａｖベクター
KR102572449B1 (ko) 곤충 세포에서 생산된 더욱 향상된 aav 벡터
US7105345B2 (en) Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
JP4573437B2 (ja) アデノ随伴ウイルス血清型１核酸配列、ベクターおよび同一物を含有する宿主細胞
Park et al. Scalable production of adeno‐associated virus type 2 vectors via suspension transfection
CN106884014B (zh) 腺相关病毒反向末端重复序列突变体及其应用
JP2024515902A (ja) 組織向性が改善されたアデノ随伴ウイルスベクターカプシド
WO2023025920A1 (en) Insect cell-produced high potency aav vectors with cns-tropism
HK40020047A (en) Capsid
CN120209091A (zh) 靶向肝细胞的重组aav衣壳蛋白及其应用
Rizzo Cloning in the cathepsin gene region of baculovirus to support rAAV production

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20191017

EEER Examination request

Effective date: 20210510

EEER Examination request

Effective date: 20210510

MPN Maintenance fee for patent paid

Free format text: FEE DESCRIPTION TEXT: MF (PATENT, 9TH ANNIV.) - STANDARD

Year of fee payment: 9

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250502

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250502