TW201704253A - 殼體 - Google Patents

殼體 Download PDF

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Publication number
TW201704253A
TW201704253A TW105114580A TW105114580A TW201704253A TW 201704253 A TW201704253 A TW 201704253A TW 105114580 A TW105114580 A TW 105114580A TW 105114580 A TW105114580 A TW 105114580A TW 201704253 A TW201704253 A TW 201704253A
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Taiwan
Prior art keywords
aav
sequence
vector
protein
amino acid
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TW105114580A
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English (en)
Chinese (zh)
Inventor
阿密特 納斯瓦尼
艾利森 丹恩
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Ucl企業有限公司
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Publication of TW201704253A publication Critical patent/TW201704253A/zh

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • 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)
TW105114580A 2015-05-11 2016-05-11 殼體 TW201704253A (zh)

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
TW201704253A true TW201704253A (zh) 2017-02-01

Family

ID=53489438

Family Applications (1)

Application Number Title Priority Date Filing Date
TW105114580A TW201704253A (zh) 2015-05-11 2016-05-11 殼體

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 (48)

* 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
IL314171A (en) 2015-09-28 2024-09-01 Univ Florida 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
EP3562514B1 (en) 2016-12-30 2026-04-29 The Trustees Of The University Of Pennsylvania Gene therapy for treating wilson's disease
JP2020530307A (ja) * 2017-06-30 2020-10-22 インティマ・バイオサイエンス,インコーポレーテッド 遺伝子治療のためのアデノ随伴ウイルスベクター
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
WO2019195423A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
WO2019195444A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
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
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
SG11202106855YA (en) 2019-01-04 2021-07-29 Ultragenyx Pharmaceutical Inc Gene therapy constructs for treating wilson disease
AR118465A1 (es) * 2019-03-21 2021-10-06 Stridebio Inc 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
ES2928689T3 (es) 2019-04-12 2022-11-22 Freeline Therapeutics Ltd Sistema de plásmidos
WO2020208379A1 (en) 2019-04-12 2020-10-15 Freeline Therapeutics Limited Plasmid system
EP4006160A4 (en) * 2019-07-12 2023-06-28 Gene Therapy Research Institution Co., Ltd. Adeno-associated virus virion for gene transfer to human liver
WO2021030764A1 (en) * 2019-08-14 2021-02-18 University Of Florida Research Foundation, Incorporated Aav capsid variants for gene therapy
WO2021041489A1 (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
KR20220083708A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 Aav 전달 카세트
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
EP4110792A4 (en) * 2020-02-25 2024-08-21 Children's Medical Research Institute POLYPEPTIDES AND CAPSIDIC VECTORS OF ADENO-ASSOCIATED VIRUSES
WO2022003211A1 (en) * 2020-07-03 2022-01-06 Genethon Method for engineering novel hybrid aav capsids through hypervariable regions swapping
CN116121275B (zh) * 2020-07-29 2024-11-29 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
MX2023002016A (es) 2020-08-19 2023-06-26 Sarepta Therapeutics Inc Vectores de virus adenoasociados para el tratamiento del sindrome de rett.
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
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
JP2023547832A (ja) 2020-10-28 2023-11-14 リジェネックスバイオ インコーポレイテッド 眼の適応症に対するベクター化抗TNF-α抗体
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
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
WO2023215806A2 (en) 2022-05-03 2023-11-09 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
WO2023215807A1 (en) 2022-05-03 2023-11-09 Regenxbio Inc. VECTORIZED ANTI-TNF-α INHIBITORS FOR OCULAR INDICATIONS
GB202214328D0 (en) 2022-09-29 2022-11-16 Freeline Therapeutics Ltd Method
AU2024271004A1 (en) 2023-05-16 2026-01-15 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
EP1030861A4 (en) 1997-10-31 2001-09-05 Maxygen Inc MODIFICATION OF VIRUSTROPISMIUS AND THE ECONOMIC SPECTRUM BY VIRAL GENOM MIXING
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
SG10202108118RA (en) 2001-11-13 2021-08-30 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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
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
PL2357189T3 (pl) 2003-06-19 2017-08-31 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
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
HUE054805T2 (hu) 2003-09-30 2021-09-28 Univ Pennsylvania Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
AU2005316476A1 (en) 2004-12-15 2006-06-22 University Of Florida Research Foundation, Inc. Chimeric vectors
CA2610164A1 (en) 2005-05-31 2006-12-07 Neurologix, Inc. Novel glutamic acid decarboxylase (gad) chimera and methods of use
US7867484B2 (en) 2006-01-27 2011-01-11 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
EP2069359B1 (en) 2006-08-21 2014-11-12 Genentech, Inc. Aza-benzothiophenyl compounds and methods of use
HUE030719T2 (en) 2007-04-09 2017-05-29 Univ Florida Raav vector compositions having tyrosine-modified capsid proteins and methods for 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
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
US8889641B2 (en) 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
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
CA2853482C (en) 2011-10-28 2020-05-19 Joshua Grieger 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
AU2013336601B2 (en) 2012-10-26 2018-01-25 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
EP2968612B1 (en) 2013-03-14 2018-02-14 Asklepios Biopharmaceutical, Inc. Modified soluble vegf receptor-1 genes and vectors for gene therapy
AU2014227766B2 (en) 2013-03-15 2018-10-04 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding AAV vectors
KR102298579B1 (ko) 2013-05-21 2021-09-07 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
PT3024498T (pt) 2013-07-22 2020-03-06 Childrens Hospital Philadelphia Variante aav e composições, métodos e usos para transferência genética em células, órgãos e tecidos
RS63942B1 (sr) 2013-08-27 2023-02-28 Res Inst Nationwide Childrens Hospital Proizvodi i postupci za lečenje amiotrofične lateralne skleroze
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
WO2015126972A1 (en) 2014-02-21 2015-08-27 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
ES2768763T3 (es) 2014-03-04 2020-06-23 Univ Florida Vectores rAAV mejorados y métodos para la transducción de fotorreceptores y células EPR
CA3205555A1 (en) 2014-04-25 2015-10-29 Genethon Treatment of hyperbilirubinemia
EP3137497B2 (en) 2014-05-02 2025-03-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
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
EP4344741A3 (en) 2014-11-21 2024-08-28 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)
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
ES2860712T3 (es) 2015-03-24 2021-10-05 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
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
PT3313991T (pt) 2015-06-23 2024-09-27 Childrens Hospital Philadelphia Fator ix modificado e composições, métodos e utilizações para transferência de genes para células, órgãos e tecidos
US20180216133A1 (en) 2015-07-17 2018-08-02 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
IL314171A (en) 2015-09-28 2024-09-01 Univ Florida 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
EP3368054A4 (en) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. REGULATORY EXPRESSION USING THE ADENO-ASSOCIATED VIRUS (AAV)
WO2017075338A2 (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
PE20181156A1 (es) 2015-11-05 2018-07-19 Bamboo Therapeutics Inc Genes de ataxia de friedreich modificados y vectores para terapia genica
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
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
EP3875593A1 (en) 2015-12-11 2021-09-08 California Institute of Technology Targeting peptides for directing adeno-associated viruses (aavs)
BR112018011881A2 (pt) 2015-12-14 2018-12-04 The University Of North Carolina At Chapel Hill proteínas capsidiais modificadas para liberação aumentada de vetores de parvovírus
JP6947739B2 (ja) 2016-02-16 2021-10-13 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 既存のヒト中和抗体に対して耐性である新規組換えアデノ随伴ウイルスカプシド
CA3018076A1 (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
US20190224339A1 (en) 2016-04-29 2019-07-25 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
CA3027784A1 (en) 2016-06-16 2017-12-21 Charite - Universitatsmedizin Berlin Neuropeptide-expressing vectors and methods for the treatment of epilepsy
AU2017301600B2 (en) 2016-07-26 2022-08-04 Biomarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
SG10201913002QA (en) 2016-08-15 2020-03-30 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
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
CA3040483A1 (en) 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
CN111344396A (zh) 2016-10-27 2020-06-26 因提玛生物科学公司 制备遗传修饰细胞的病毒方法
CA3052829A1 (en) 2017-02-15 2018-08-23 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature
WO2018162748A1 (en) 2017-03-10 2018-09-13 Genethon Treatment of glycogen storage disease iii
IL316259A (en) 2017-03-15 2024-12-01 Univ North Carolina Chapel Hill Polyploid gland associated with virus vectors and methods for preparing and using the same.
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
JP2020518259A (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. ハンチントン病治療組成物および方法
SG11201909870SA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
KR20250030036A (ko) 2017-06-07 2025-03-05 스파크 테라퓨틱스, 인코포레이티드 개선된 세포 트랜스펙션 및/또는 rAAV 벡터 생산을 위한 증진제
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
SG11201913157RA (en) 2017-06-30 2020-01-30 Spark Therapeutics Inc Aav vector column purification methods
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
KR20240160648A (ko) 2017-08-01 2024-11-11 스파크 테라퓨틱스, 인코포레이티드 인자 viii (fviii) 유전자 요법 방법
TWI832036B (zh) 2017-08-03 2024-02-11 美商航海家醫療公司 用於aav之遞送之組合物及方法
EP3676284A1 (en) 2017-08-29 2020-07-08 Ruprecht-Karls-Universität 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
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

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