JP6619454B2 - キャプシド - Google Patents

キャプシド Download PDF

Info

Publication number
JP6619454B2
JP6619454B2 JP2017558723A JP2017558723A JP6619454B2 JP 6619454 B2 JP6619454 B2 JP 6619454B2 JP 2017558723 A JP2017558723 A JP 2017558723A JP 2017558723 A JP2017558723 A JP 2017558723A JP 6619454 B2 JP6619454 B2 JP 6619454B2
Authority
JP
Japan
Prior art keywords
aav
sequence
capsid
sequences
capsid protein
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
JP2017558723A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018520997A5 (enExample
JP2018520997A (ja
Inventor
ナスワニ、アミット
デイン、アリソン
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 JP2018520997A publication Critical patent/JP2018520997A/ja
Publication of JP2018520997A5 publication Critical patent/JP2018520997A5/ja
Application granted granted Critical
Publication of JP6619454B2 publication Critical patent/JP6619454B2/ja
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)
JP2017558723A 2015-05-11 2016-05-10 キャプシド Active JP6619454B2 (ja)

Applications Claiming Priority (3)

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2019206191A Division JP2020096587A (ja) 2015-05-11 2019-11-14 キャプシド

Publications (3)

Publication Number Publication Date
JP2018520997A JP2018520997A (ja) 2018-08-02
JP2018520997A5 JP2018520997A5 (enExample) 2019-06-13
JP6619454B2 true JP6619454B2 (ja) 2019-12-11

Family

ID=53489438

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2017558723A Active JP6619454B2 (ja) 2015-05-11 2016-05-10 キャプシド
JP2019206191A Pending JP2020096587A (ja) 2015-05-11 2019-11-14 キャプシド

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2019206191A Pending JP2020096587A (ja) 2015-05-11 2019-11-14 キャプシド

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)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020096587A (ja) * 2015-05-11 2020-06-25 ユーシーエル ビジネス リミテッド キャプシド

Families Citing this family (46)

* 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
CA2996420A1 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
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
EP3774853A1 (en) * 2018-03-30 2021-02-17 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
SG11202009451VA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
CN112272672A (zh) 2018-04-03 2021-01-26 斯特里迪比奥公司 抗体逃避性病毒载体
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
PT3906066T (pt) 2019-01-04 2024-02-06 Ultragenyx Pharmaceutical Inc Constructos de terapia génica para o tratamento da doença de wilson
SG11202110146VA (en) * 2019-03-21 2021-10-28 Stridebio Inc Recombinant adeno-associated virus vectors
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
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
PL3722434T3 (pl) 2019-04-12 2022-12-12 Freeline Therapeutics Limited Układ plazmidowy
US20220265853A1 (en) * 2019-07-12 2022-08-25 Gene Therapy Research Institution Co., Ltd. Adeno-associated virus virion for gene transfer to human liver
AU2020330121B2 (en) * 2019-08-14 2025-10-30 University Of Florida Research Foundation, Incorporated AAV capsid variants for gene therapy
CA3151087A1 (en) * 2019-08-29 2021-03-04 The Board Of Trustees Of The Leland Stanford Junior University Adeno-associated viral vectors for crossing the human blood brain barrier
EP4045525A1 (en) 2019-10-17 2022-08-24 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
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
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
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
WO2022060916A1 (en) 2020-09-15 2022-03-24 Regenxbio Inc. Vectorized antibodies for anti-viral therapy
EP4213890A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized lanadelumab and administration thereof
GB202016254D0 (en) 2020-10-13 2020-11-25 Freeline Therapeutics Ltd Plasmid system
EP4236999A1 (en) 2020-10-28 2023-09-06 RegenxBio Inc. Vectorized anti-tnf-alfa antibodies for ocular indications
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
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
EP4518972A2 (en) 2022-05-03 2025-03-12 RegenxBio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
TW202417633A (zh) 2022-05-03 2024-05-01 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抑制劑
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
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
AU1449499A (en) 1997-10-31 1999-05-24 Maxygen, Incorporated Modification of virus tropism and host range by viral genome shuffling
CA2348382C (en) 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Chimeric parvovirus 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
AU4565401A (en) * 2000-03-14 2001-09-24 Thomas Jefferson University Production of chimeric capsid vectors
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
NZ618298A (en) 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
JP4810062B2 (ja) 2001-12-17 2011-11-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス（ａａｖ）血清型８の配列
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
EP2657248B1 (en) 2003-06-19 2017-03-22 Genzyme Corporation AAV virions with decreased immunoreactivity and uses therefor
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
HUE035567T2 (en) 2003-09-30 2018-05-28 Univ Pennsylvania Adeno-associated virus (AAV) clusters, sequences, vectors and applications containing them
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
NZ555830A (en) 2004-12-15 2009-01-31 Univ North Carolina Chimeric vectors
EP1893754A4 (en) 2005-05-31 2009-01-21 Neurologix Inc NEW GLUTAMIC ACID DECARBOXYLASE (GAD) CHIMESES AND USE PROCESSES
US7867484B2 (en) 2006-01-27 2011-01-11 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
AU2007286807B2 (en) 2006-08-21 2013-03-21 Genentech, Inc. Aza-benzothiophenyl compounds and methods of use
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
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
WO2008145400A2 (en) * 2007-05-31 2008-12-04 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
PL2900686T3 (pl) 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
DK2911687T3 (da) 2012-10-26 2019-05-13 Univ Brussel Vrije Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf
ES2667209T3 (es) 2013-03-14 2018-05-10 Asklepios Biopharmaceutical, Inc. Genes del receptor-1 de VEGF soluble modificado y vectores para terapia génica
WO2014144229A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav 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
SI3024498T1 (sl) 2013-07-22 2020-07-31 The Children's Hospital Of Philadelphia Različica AAV in sestavki, postopki in uporabe za prenos genov v celice, organe in tkiva
EP4219727A3 (en) 2013-08-27 2023-09-06 Research Institute at Nationwide Children's Hospital Products and methods for treatment of amyotrophic lateral sclerosis
EP3459965B1 (en) * 2013-10-11 2020-12-02 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
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
JP6730193B2 (ja) 2014-04-25 2020-07-29 ジェネトン 高ビリルビン血症の処置
PT3137497T (pt) 2014-05-02 2021-07-12 Genzyme Corp Vetores de aav para terapia genética na retina e snc
US10577627B2 (en) 2014-06-09 2020-03-03 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
CA2967393A1 (en) 2014-11-21 2016-05-26 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
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
EP3262162A4 (en) 2015-02-23 2018-08-08 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
JP7095994B2 (ja) 2015-03-02 2022-07-05 アドヴェラム バイオテクノロジーズ， インコーポレイテッド ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法
CA2979065A1 (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
IL309741A (en) 2015-06-23 2024-02-01 Childrens Hospital 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
US20190000940A1 (en) 2015-07-31 2019-01-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
CA2996420A1 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
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)
CA3002406A1 (en) 2015-10-29 2017-05-04 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
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
SG10201912763QA (en) 2015-11-05 2020-02-27 Bamboo Therapeutics Inc Modified friedreich ataxia genes and vectors for gene therapy
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
CA3007495C (en) 2015-12-11 2023-04-11 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (aavs)
CA3008013A1 (en) 2015-12-14 2017-06-22 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
CN109415704B (zh) 2016-02-16 2022-02-25 利兰斯坦福初级大学董事会 对先已存在的人中和抗体有抗性的新型重组腺相关病毒衣壳
AU2017234929B2 (en) 2016-03-18 2024-05-02 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
WO2017189963A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
IL262784B2 (en) 2016-05-18 2023-10-01 Voyager Therapeutics Inc modulatory polynucleotides
WO2017216301A1 (en) 2016-06-16 2017-12-21 Charité Berlin Neuropeptide-expressing vectors and methods for the treatment of epilepsy
EP3491008A2 (en) 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
NZ791267A (en) 2016-08-15 2025-11-28 Genzyme Corp Methods for detecting AAV
CN110121356A (zh) 2016-09-02 2019-08-13 星火治疗股份有限公司 治疗cns疾病的方法和载体
EP3293259A1 (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
EP3293203A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
GB2573663B (en) 2016-10-27 2023-04-26 Intima Bioscience Inc Viral methods of making genetically modified cells
EP3583220A4 (en) 2017-02-15 2021-07-07 The University of North Carolina at Chapel Hill PROCEDURES AND COMPOSITIONS FOR GENERAL TRANSFER IN THE VASCULAR SYSTEM
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
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
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
PE20251181A1 (es) 2017-06-07 2025-04-23 Spark Therapeutics Inc Agentes potenciadores para la transfeccion celular mejorada y/o la produccion del vector raav
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.
CN120989074A (zh) 2017-07-31 2025-11-21 映像生物有限公司 眼部疾病的细胞模型及用于眼部疾病的疗法
SG11202000650YA (en) 2017-08-01 2020-02-27 Spark Therapeutics Inc Factor viii (fviii) gene therapy methods
AU2018311069A1 (en) 2017-08-03 2020-02-20 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
AU2018325362B2 (en) 2017-08-29 2023-07-20 Ruprecht-Karls-Universität 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
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

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020096587A (ja) * 2015-05-11 2020-06-25 ユーシーエル ビジネス リミテッド キャプシド

Also Published As

Publication number Publication date
CA2985945A1 (en) 2016-11-17
EP3250239A1 (en) 2017-12-06
CN107864653A (zh) 2018-03-30
PL3250239T3 (pl) 2019-05-31
US20250127922A1 (en) 2025-04-24
KR20180019543A (ko) 2018-02-26
CN107864653B (zh) 2022-06-21
CA2985945C (en) 2022-09-06
EP3511021A1 (en) 2019-07-17
TR201901582T4 (tr) 2019-02-21
AU2016261454B2 (en) 2019-07-11
HUE042693T2 (hu) 2019-07-29
JP2018520997A (ja) 2018-08-02
US20180111965A1 (en) 2018-04-26
US20200079821A1 (en) 2020-03-12
TW201704253A (zh) 2017-02-01
AU2016261454A1 (en) 2017-11-23
GB201508026D0 (en) 2015-06-24
DK3250239T3 (en) 2019-03-11
US12138318B2 (en) 2024-11-12
ES2711349T3 (es) 2019-05-03
US10414803B2 (en) 2019-09-17
WO2016181123A1 (en) 2016-11-17
LT3250239T (lt) 2019-03-25
PT3250239T (pt) 2019-02-19
EP3250239B1 (en) 2018-12-12
HRP20190442T1 (hr) 2019-05-03
JP2020096587A (ja) 2020-06-25

Similar Documents

Publication Publication Date Title
JP6619454B2 (ja) キャプシド
US20220331409A1 (en) Factor ix gene therapy
Hauck et al. Generation and characterization of chimeric recombinant AAV vectors
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
CN114127088A (zh) 新的腺相关病毒(aav)变体及其在基因治疗中的用途
JP2025178242A (ja) Ａａｖ５の単離修飾ｖｐ１カプシドタンパク質
Wang et al. High-density recombinant adeno-associated viral particles are competent vectors for in vivo transduction
JP7371954B2 (ja) 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
JP2023509443A (ja) 改善されたａａｖ－ａｂｃｄ１コンストラクトならびに副腎白質ジストロフィー（ａｌｄ）および／または副腎脊髄ニューロパチー（ａｍｎ）の処置または予防のための使用
JP2019216667A (ja) 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
AU2018394287A1 (en) Modified viral vectors and methods of making and using the same
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
HK40020047A (en) Capsid
RU2825667C2 (ru) Выделенный модифицированный белок VPI капсида аденоассоциированного вируса 9 серотипа (AAV9), капсид и вектор на его основе
CN117836312A (zh) 分离的修饰的aav9衣壳蛋白vp1
CN117881689A (zh) 分离的修饰的aav5衣壳蛋白vp1
EA048714B1 (ru) Выделенный модифицированный белок vp1 капсида аденоассоциированного вируса 9 серотипа (aav9), капсид и вектор на его основе

Legal Events

Date Code Title Description
RD01 Notification of change of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7426

Effective date: 20180305

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20180305

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190510

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20190510

A871 Explanation of circumstances concerning accelerated examination

Free format text: JAPANESE INTERMEDIATE CODE: A871

Effective date: 20190510

A975 Report on accelerated examination

Free format text: JAPANESE INTERMEDIATE CODE: A971005

Effective date: 20190522

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20190604

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190903

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20191015

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20191114

R150 Certificate of patent or registration of utility model

Ref document number: 6619454

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250