AU780231B2 - Virus vectors and methods of making and administering the same - Google Patents

Virus vectors and methods of making and administering the same Download PDF

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Publication number
AU780231B2
AU780231B2 AU19111/00A AU1911100A AU780231B2 AU 780231 B2 AU780231 B2 AU 780231B2 AU 19111/00 A AU19111/00 A AU 19111/00A AU 1911100 A AU1911100 A AU 1911100A AU 780231 B2 AU780231 B2 AU 780231B2
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aav
capsid
cell
virus particle
nucleic acid
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AU19111/00A
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AU1911100A (en
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Joseph E. Rabinowitz
Richard Jude Samulski
Weidong Xiao
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University of North Carolina at Chapel Hill
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University of North Carolina at Chapel Hill
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    • 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
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    • A61K2039/525Virus
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    • C12N2750/14011Parvoviridae
    • C12N2750/14041Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011Parvoviridae
    • C12N2750/14211Erythrovirus, e.g. B19 virus
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    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/80Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
    • C12N2810/85Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian
    • C12N2810/854Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian from hormones
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10STECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10S977/00Nanotechnology
    • Y10S977/70Nanostructure
    • Y10S977/788Of specified organic or carbon-based composition
    • Y10S977/802Virus-based particle
    • Y10S977/803Containing biological material in its interior
    • Y10S977/804Containing nucleic acid

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AU19111/00A 1998-11-10 1999-11-10 Virus vectors and methods of making and administering the same Expired AU780231B2 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US10784098P 1998-11-10 1998-11-10
US60/107840 1998-11-10
US12365199P 1999-03-10 1999-03-10
US60/123651 1999-03-10
PCT/US1999/026505 WO2000028004A1 (en) 1998-11-10 1999-11-10 Virus vectors and methods of making and administering the same

Publications (2)

Publication Number Publication Date
AU1911100A AU1911100A (en) 2000-05-29
AU780231B2 true AU780231B2 (en) 2005-03-10

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AU19111/00A Expired AU780231B2 (en) 1998-11-10 1999-11-10 Virus vectors and methods of making and administering the same

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US (4) US6491907B1 (https=)
EP (1) EP1135468B1 (https=)
JP (2) JP2002538770A (https=)
AT (1) ATE454445T1 (https=)
AU (1) AU780231B2 (https=)
CA (1) CA2348382C (https=)
DE (1) DE69941905D1 (https=)
ES (1) ES2340230T3 (https=)
WO (1) WO2000028004A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2001255575B2 (en) * 2000-04-28 2006-08-31 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids

Families Citing this family (475)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE19827457C1 (de) * 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
AU780231B2 (en) * 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
US7314912B1 (en) 1999-06-21 2008-01-01 Medigene Aktiengesellschaft AAv scleroprotein, production and use thereof
DE19933288A1 (de) 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
DE19933719A1 (de) * 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
US7115391B1 (en) * 1999-10-01 2006-10-03 Genovo, Inc. Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
CA2373110A1 (en) * 2000-03-14 2001-09-20 Neurologix, Inc. Production of chimeric capsid vectors
US7638120B2 (en) * 2000-03-14 2009-12-29 Thomas Jefferson University High transgene expression of a pseudotyped adeno-associated virus type
US7201898B2 (en) * 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
WO2002000924A2 (en) * 2000-06-28 2002-01-03 Institut National De La Recherche Scientifique Viral phospholipase a2 enzymes, anti-viral agents and methods of use
US7749492B2 (en) * 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
WO2002053703A2 (en) * 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
DE10120265A1 (de) * 2001-04-25 2002-11-14 Deutsches Krebsforsch AAV-Helferplasmide zur Helfervirus-freien Verpackung und Pseudotypisierung von AAV-Vektoren
US8067156B2 (en) * 2001-05-31 2011-11-29 The Rockefeller University Method for generating replication defective viral vectors that are helper free
US6859916B1 (en) 2001-06-03 2005-02-22 Cadence Design Systems, Inc. Polygonal vias
US6905678B2 (en) * 2001-07-07 2005-06-14 Crucell Holland B.V. Gene delivery vectors with cell type specificity for mesenchymal stem cells
US8241622B2 (en) * 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
JP2005503418A (ja) * 2001-09-14 2005-02-03 ユニベルシテ ピエール エ マリー キュリー(パリ シズエム) 治療的ワクチン接種法、hiv逆転写酵素の突然変異ペプチドならびにワクチン接種及び診断目的のためのそれらの使用
DE10210139A1 (de) * 2002-03-07 2003-10-23 Medigene Ag Helferkonstrukte für die Herstellung hybrider rAAV-Partikel unterschiedlicher AAV-Serotypen
AU2003223497A1 (en) * 2002-04-05 2003-10-27 Centocor, Inc. Asthma-related anti-il-13 immunoglobulin derived proteins, compositions, methods and uses
WO2003093440A2 (en) * 2002-04-30 2003-11-13 University Of Florida Methods and compositions for expressing a nucleic acid in a dendritic cell
AU2003274397A1 (en) * 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
KR100535325B1 (ko) * 2002-09-24 2005-12-09 한국생명공학연구원 Aav 유전자 전달체 제조용 헬퍼 플라스미드
US20040208847A1 (en) * 2003-03-28 2004-10-21 Fabienne Rolling Method and vectors for selectively transducing retinal pigment epithelium cells
SI1633772T1 (sl) * 2003-06-19 2016-06-30 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
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
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US7972593B2 (en) * 2004-06-10 2011-07-05 Saint Louis University Delivery of therapeutic agents to the bone
US20100132058A1 (en) 2004-07-23 2010-05-27 Diatchenko Luda B Methods and materials for determining pain sensitivity and predicting and treating related disorders
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
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
WO2007084773A2 (en) * 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
CA2638913A1 (en) 2006-02-10 2007-09-07 The University Of Cincinnati Phosphatase inhibitor protein-1 as a regulator of cardiac function
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
ES2535877T3 (es) 2006-06-19 2015-05-18 Asklepios Biopharmaceutical, Inc. Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica
WO2008054713A2 (en) * 2006-10-30 2008-05-08 Thomas Jefferson University Tissue specific gene therapy treatment
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
EP2012122A1 (en) * 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
WO2009105135A1 (en) 2008-02-19 2009-08-27 Celladon Corporation Compositions for enhanced uptake of viral vectors in the myocardium
WO2009108274A2 (en) * 2008-02-26 2009-09-03 The University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
AU2009332883B2 (en) * 2008-12-22 2015-05-21 Oncos Therapeutics Oy Oncolytic adenoviral vectors and methods and uses related thereto
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US20120093775A1 (en) 2009-03-27 2012-04-19 Proyecto De Biomedicina Cima, S.L. Methods and compositions for the treatment of cirrhosis and liver fibrosis
EP2425000B1 (en) * 2009-04-30 2019-02-20 The Trustees Of The University Of Pennsylvania Compositions for targeting conducting airway cells comprising adeno-associated virus constructs
WO2011032100A1 (en) 2009-09-11 2011-03-17 Government Of The U.S.A., As Represented By The Secretary, Department Of Health And Human Services Inhibitors of kshv vil6 and human il6
BR112012010755A2 (pt) 2009-11-05 2015-09-22 Proyecto Biomedicina Cima Sl constructo de gene que permite a expressão hepato-específica induzível de um polinucleotídeo de interesse em resposta a um agente indutor, vetor, genoma viral recombinante, vírion, método in vitro para a expressão de um polinucleotídeo de interesse em uma célula de origem hepática, composição farmacêutica e operador-promotor bi-direcional induzível adequado para a expressão hepato-específica induzível de dois polinucleotídeos de interesse por um agente indutor
EP2524037B1 (en) 2010-01-12 2018-05-16 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
WO2011094198A1 (en) 2010-01-28 2011-08-04 The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
EP2534172A2 (en) * 2010-02-12 2012-12-19 The U.S.A. as represented by the Secretary, Department of Health and Human Services Compositions and methods for preventing or treating a human parvovirus infection
DK2542260T3 (en) * 2010-03-05 2016-06-06 Intervet Int Bv Recombinantly weakening parvovirus
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
BR112012026095A2 (pt) 2010-04-14 2015-09-15 Emd Millipore Corp métodos de produção de estoques de vírus de alta pureza e altos títulos e métodos de uso dos mesmos.
EP2397542A1 (en) * 2010-06-17 2011-12-21 Deutsches Krebsforschungszentrum Modified parvovirus having enhanced anti-tumour efficacy
AU2011276328C1 (en) 2010-07-06 2016-01-21 Novartis Ag Norovirus derived immunogenic compositions and methods
EP2605798A1 (en) 2010-07-12 2013-06-26 Universidad Autònoma de Barcelona Gene therapy composition for use in diabetes treatment
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
EP2675484B1 (en) 2011-02-14 2018-05-30 The Children's Hospital of Philadelphia Improved aav8 vector with enhanced functional activity and methods of use thereof
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
JP6198278B2 (ja) 2011-09-08 2017-09-20 ユニキュアー アイピー ビー.ブイ. Aav調製物からの混入ウイルスの除去
WO2013052915A2 (en) 2011-10-05 2013-04-11 Genelux Corporation Method for detecting replication or colonization of a biological therapeutic
US20140087362A1 (en) 2012-03-16 2014-03-27 Aladar A. Szalay Methods for assessing effectiveness and monitoring oncolytic virus treatment
WO2013158265A1 (en) 2012-04-20 2013-10-24 Genelux Corporation Imaging methods for oncolytic virus therapy
WO2013170078A1 (en) 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
US20150182637A1 (en) 2012-06-21 2015-07-02 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
EP2900686B1 (en) 2012-09-28 2020-06-10 The University of North Carolina At Chapel Hill Aav vectors targeted to oligodendrocytes
US20140140959A1 (en) 2012-10-05 2014-05-22 Aladar A. Szalay Energy Absorbing-Based Diagnostic and Therapeutic Methods Employing Nucleic Acid Molecules Encoding Chromophore-Producing Enzymes
ES2667209T3 (es) 2013-03-14 2018-05-10 Asklepios Biopharmaceutical, Inc. Genes del receptor-1 de VEGF soluble modificado y vectores para terapia génica
WO2014143932A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
ES2926774T3 (es) 2013-03-15 2022-10-28 Univ North Carolina Chapel Hill Métodos y composiciones para vectores AAV de doble unión de glicano
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2014168953A1 (en) 2013-04-08 2014-10-16 University Of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
EP3003391B1 (en) 2013-05-31 2021-09-22 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
US20160122727A1 (en) 2013-06-13 2016-05-05 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
LT3013855T (lt) 2013-06-24 2021-01-25 Xiao, Weidong Mutavusio viii faktoriaus kompozicijos ir metodai
AU2014305218B2 (en) 2013-08-05 2020-01-16 Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum Recombinant AAV-Crumbs homologue composition and methods for treating LCA-8 and progressive RP
WO2015048534A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
WO2015044292A1 (en) 2013-09-26 2015-04-02 Universitat Autonoma De Barcelona Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease
CN105745326A (zh) 2013-10-24 2016-07-06 优尼科Ip有限公司 用于基因治疗神经疾病的aav-5假型载体
WO2015077717A1 (en) 2013-11-25 2015-05-28 The Broad Institute Inc. Compositions and methods for diagnosing, evaluating and treating cancer by means of the dna methylation status
WO2015085147A1 (en) 2013-12-05 2015-06-11 The Broad Institute Inc. Polymorphic gene typing and somatic change detection using sequencing data
KR20230076867A (ko) 2013-12-20 2023-05-31 더 브로드 인스티튜트, 인코퍼레이티드 신생항원 백신과의 병용 요법
US10238700B2 (en) 2014-01-02 2019-03-26 Genelux Corporation Oncolytic virus adjunct therapy with agents that increase virus infectivity
SMT202000531T1 (it) 2014-03-09 2020-11-10 Univ Pennsylvania Composizioni utili in trattamento di deficit di ornitina transcarbamilasi(otc)
EP3800191B1 (en) 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
BR112016019940A2 (pt) 2014-03-21 2017-10-24 Univ Leland Stanford Junior edição de genoma sem nucleases
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
EP3134431B1 (en) 2014-04-25 2021-04-07 The Trustees Of The University Of Pennsylvania Ldlr variants and their use in compositions for reducing cholesterol levels
ES2833230T3 (es) 2014-05-13 2021-06-14 Univ Pennsylvania Composiciones que comprenden AVV que expresan construcciones y usos de doble anticuerpo del mismo
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
EP3194430A1 (en) 2014-09-16 2017-07-26 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
BR112017007765B1 (pt) 2014-10-14 2023-10-03 Halozyme, Inc Composições de adenosina deaminase-2 (ada2), variantes do mesmo e métodos de usar o mesmo
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
EP4344741A3 (en) 2014-11-21 2024-08-28 The University of North Carolina at Chapel Hill Aav vectors targeted to the central nervous system
HUE046643T2 (hu) * 2014-11-28 2021-11-29 Uniqure Ip Bv DNS szennyezõdések parvovirális virionokat tartalmazó készítményben
US10975442B2 (en) 2014-12-19 2021-04-13 Massachusetts Institute Of Technology Molecular biomarkers for cancer immunotherapy
EP3234130B1 (en) 2014-12-19 2020-11-25 The Broad Institute, Inc. Methods for profiling the t-cell- receptor repertoire
US11033638B2 (en) 2015-01-07 2021-06-15 Universität Autonoma De Barcelona Single-vector gene construct comprising insulin and glucokinase genes
US10907176B2 (en) 2015-01-14 2021-02-02 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
US11021519B2 (en) 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
EP3268044A2 (en) 2015-03-11 2018-01-17 The Broad Institute Inc. Prmt5 inhibitors for the treatment of cancer with reduced mtap activty
JP6836999B2 (ja) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California アデノ随伴ウイルス変異体及びその使用方法
EP3288594B1 (en) 2015-04-27 2022-06-29 The Trustees of The University of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
JP6805174B2 (ja) 2015-05-12 2020-12-23 アメリカ合衆国 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むaav分離株及び融合タンパク質
EA201792500A1 (ru) 2015-05-13 2018-04-30 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Aav-опосредованная экспрессия антител против гриппа и способы их использования
IL294183B2 (en) 2015-05-20 2023-10-01 Dana Farber Cancer Inst Inc shared neoantigens
TW202241500A (zh) 2015-06-09 2022-11-01 美商博德研究所有限公司 用於贅瘤疫苗之調配物及其製備方法
US20160361380A1 (en) 2015-06-12 2016-12-15 Nymox Corporation Combination compositions for treating disorders requiring removal or destruction of unwanted cellular proliferations
US10676735B2 (en) 2015-07-22 2020-06-09 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
GB2592821B (en) 2015-07-31 2022-01-12 Univ Minnesota Modified cells and methods of therapy
WO2017024198A1 (en) 2015-08-06 2017-02-09 The Trustees Of The University Of Pennsylvania Glp-1 and use thereof in compositions for treating metabolic diseases
US20190000943A1 (en) * 2015-08-13 2019-01-03 Aavet Therapeutics, Llc Aav6 vectors for immunotherapy
WO2017035416A2 (en) 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
CA2995733A1 (en) 2015-08-31 2017-03-09 The Trustees Of The University Of Pennsylvania Aav-epo for treating companion animals
CN108291216B (zh) 2015-09-24 2022-09-16 宾夕法尼亚州大学信托人 用于治疗补体介导的疾病的组合物和方法
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
US11273227B2 (en) 2015-10-09 2022-03-15 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating Stargardt's disease and other ocular disorders
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
CN108779167A (zh) 2015-10-28 2018-11-09 宾夕法尼亚州大学信托人 鞘内施用腺伴随病毒载体用于基因治疗
SG11201802408RA (en) 2015-11-05 2018-05-30 Bamboo Therapeutics Inc Modified friedreich ataxia genes and vectors for gene therapy
KR102787119B1 (ko) 2015-11-30 2025-03-27 듀크 유니버시티 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법
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
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
AU2016366560A1 (en) 2015-12-11 2018-06-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
JP7061067B2 (ja) 2015-12-14 2022-04-27 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 クリグラー・ナジャー症候群の処置のための組成物
JP7406783B2 (ja) 2015-12-14 2023-12-28 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 パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質
CA3008264A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
EP3411484B1 (en) 2016-02-05 2023-10-04 Emory University Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
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
CA3011943A1 (en) 2016-02-22 2017-08-31 The University Of North Carolina At Chapel Hill Aav-idua vector for treatment of mps i-associated blindness
CA3016985C (en) 2016-03-07 2023-07-04 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
JP2019513373A (ja) 2016-03-31 2019-05-30 ネオン セラピューティクス, インコーポレイテッド ネオ抗原およびその使用方法
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
WO2017181152A2 (en) 2016-04-15 2017-10-19 Alpine Immune Sciences, Inc. Cd80 variant immunomodulatory proteins and uses thereof
MX2018012472A (es) 2016-04-15 2019-08-12 Alpine Immune Sciences Inc Proteinas inmunomoduladoras variantes de ligando icos y sus usos.
AU2017254477A1 (en) 2016-04-18 2018-11-01 Jennifer G. ABELIN Improved HLA epitope prediction
EP3449250B1 (en) 2016-04-28 2020-11-04 Indiana University Research & Technology Corporation Methods and compositions for resolving components of a virus preparation
US11364308B2 (en) 2016-05-13 2022-06-21 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
WO2017205739A1 (en) 2016-05-26 2017-11-30 University Of Iowa Research Foundation cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1
CN109415730A (zh) 2016-06-13 2019-03-01 北卡罗来纳大学教堂山分校 优化的cln1基因和表达盒以及它们的应用
US10183058B2 (en) 2016-06-17 2019-01-22 Nymox Corporation Method of preventing or reducing the progression of prostate cancer
CA2971303C (en) 2016-06-21 2026-03-03 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
WO2018009814A1 (en) 2016-07-08 2018-01-11 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving rdh12
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Cpf1に基づくゲノム編集の治療適用
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
BR112019001344A2 (pt) 2016-07-26 2019-04-30 The University Of North Carolina At Chapel Hill tolerância imunológica mediada por vetor no olho
WO2018022945A1 (en) 2016-07-28 2018-02-01 Alpine Immune Sciences, Inc. Cd112 variant immunomodulatory proteins and uses thereof
US11471488B2 (en) 2016-07-28 2022-10-18 Alpine Immune Sciences, Inc. CD155 variant immunomodulatory proteins and uses thereof
WO2018022946A1 (en) 2016-07-28 2018-02-01 Alpine Immune Sciences, Inc. Cd155 variant immunomodulatory proteins and uses thereof
US10172910B2 (en) 2016-07-28 2019-01-08 Nymox Corporation Method of preventing or reducing the incidence of acute urinary retention
AU2017302013B2 (en) 2016-07-29 2022-05-26 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
EP4485466A3 (en) * 2016-08-17 2025-04-02 The Broad Institute Inc. Novel crispr enzymes and systems
WO2018035387A1 (en) * 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
IL315358A (en) 2016-08-18 2024-11-01 Univ California CRISPR-CAS genome engineering using a modular AAV delivery system
JP7165410B2 (ja) 2016-08-19 2022-11-04 ユニバーシティ オブ フロリダ リサーチ ファウンデーション,インコーポレイティド 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物
US10532081B2 (en) 2016-09-07 2020-01-14 Nymox Corporation Method of ameliorating or preventing the worsening or the progression of symptoms of BPH
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
WO2018075978A1 (en) 2016-10-20 2018-04-26 Alpine Immune Sciences, Inc. Secretable variant immunomodulatory proteins and engineered cell therapy
US20190328846A1 (en) 2016-12-01 2019-10-31 INSERM (Institut National de la Santé et de la Recherche Médicale) Pharmaceutical compositions for the treatment of retinal degenerative diseases
KR20190088561A (ko) 2016-12-07 2019-07-26 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 인터류킨-1 수용체 길항제(IL-1RA) cDNA
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
US11549149B2 (en) 2017-01-24 2023-01-10 The Broad Institute, Inc. Compositions and methods for detecting a mutant variant of a polynucleotide
CN110546265A (zh) 2017-02-09 2019-12-06 因达普塔治疗公司 工程化自然杀伤(nk)细胞及其组合物和方法
US11965892B2 (en) 2017-02-12 2024-04-23 Biontech Us Inc. HLA-based methods and compositions and uses thereof
US11905312B2 (en) * 2017-02-15 2024-02-20 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature
JP2020510648A (ja) 2017-02-20 2020-04-09 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 家族性高コレステロール血症を処置するための遺伝子治療
EP3585883A4 (en) 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
UY37620A (es) 2017-02-28 2018-08-31 Univ Pennsylvania Vacunas contra la gripe mediadas por aav novedosas
PT3589730T (pt) 2017-02-28 2024-02-28 Univ Pennsylvania Vetor de vírus adenoassociado (aav) de clado f e suas utilizações
RU2019130004A (ru) 2017-03-01 2021-04-01 Зе Трастис Оф Зе Юниверсити Оф Пеннсильвания Генная терапия при глазных заболеваниях
US10335453B2 (en) 2017-03-01 2019-07-02 Nymox Corporation Compositions and methods for improving sexual function
US10550405B2 (en) 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
NZ756587A (en) 2017-03-15 2025-12-19 Univ North Carolina Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
SG11201907769XA (en) 2017-03-16 2019-09-27 Alpine Immune Sciences Inc Cd80 variant immunomodulatory proteins and uses thereof
KR20190141146A (ko) 2017-03-16 2019-12-23 알파인 이뮨 사이언시즈, 인코포레이티드 Pd-l2 변이체 면역조절 단백질 및 그의 용도
EP3596116B1 (en) 2017-03-16 2023-09-06 Alpine Immune Sciences, Inc. Pd-l1 variant immunomodulatory proteins and uses thereof
WO2018195555A1 (en) 2017-04-21 2018-10-25 The Board Of Trustees Of The Leland Stanford Junior University Crispr/cas 9-mediated integration of polynucleotides by sequential homologous recombination of aav donor vectors
WO2018200542A1 (en) 2017-04-24 2018-11-01 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
IL316926A (en) 2017-05-11 2025-01-01 Univ Pennsylvania Gene therapy for neuronal ceroid lipofuscinosis
EP3625344A1 (en) 2017-05-15 2020-03-25 University of Florida Research Foundation, Inc. Core master regulators of glioblastoma stem cells
IL270882B2 (en) 2017-05-24 2024-02-01 Univ Barcelona Autonoma Viral expression vectors containing the coding sequence for FIBROBLAST GROWTH FACTOR 21 (FGF21
EP3630160A1 (en) 2017-05-24 2020-04-08 Universitat Autònoma De Barcelona Viral expression construct comprising a fibroblast growth factor 21 (fgf21) coding sequence
EP3630986B1 (en) 2017-05-31 2026-03-25 The Trustees of The University of Pennsylvania Gene therapy for treating peroxisomal disorders
JP7766393B2 (ja) 2017-06-14 2025-11-10 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 眼疾患のための遺伝子療法
EP3645021A4 (en) * 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
CA3053154A1 (en) 2017-06-30 2019-01-03 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
JP7764129B2 (ja) 2017-07-10 2025-11-05 ユニキュアー アイピー ビー.ブイ. ヒトにおけるaav遺伝子療法のための手段及び方法
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
JP2020534788A (ja) 2017-08-28 2020-12-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア アデノ随伴ウイルスカプシド変異体及びその使用方法
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
WO2019060538A1 (en) 2017-09-20 2019-03-28 The Trustees Of Indiana University METHODS FOR LIPOPROTEIN RESOLUTION BY MASS SPECTROMETRY
RU2770922C2 (ru) 2017-09-20 2022-04-25 4Д Молекьюлар Терапьютикс Инк. Капсиды вариантов аденоассоциированных вирусов и методы их применения
AU2018346104B2 (en) 2017-10-03 2023-07-27 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
BR112020006661A2 (pt) 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
CN112501208A (zh) 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
JP7749319B2 (ja) 2017-10-10 2025-10-06 アルパイン イミューン サイエンシズ インコーポレイテッド Ctla-4変異型免疫調節タンパク質およびそれらの使用
US12043848B2 (en) 2017-10-16 2024-07-23 Vigeneron Gmbh AAV vectors
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
BR112020007542A2 (pt) 2017-10-18 2020-12-01 Alpine Immune Sciences, Inc. proteínas imunomoduladoras ligantes de icos variantes e composições e métodos relacionados
RU2020118342A (ru) 2017-11-07 2021-12-08 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Оптимизированные гены aga и экспрессионные кластеры и их применение
CN111770999A (zh) 2017-11-27 2020-10-13 4D分子治疗有限公司 腺相关病毒变体衣壳和用于抑制血管生成的应用
US10538571B2 (en) 2017-11-27 2020-01-21 Coda Biotherapeutics, Inc. Compositions and methods for neurological diseases
TW201930340A (zh) 2017-12-18 2019-08-01 美商尼恩醫療公司 新抗原及其用途
EP3727468A4 (en) 2017-12-19 2021-09-22 Akouos, Inc. AAV-MEDIATED THERAPEUTIC ANTIBODIES DELIVERY TO THE INTERNAL EAR
AU2019205273B2 (en) 2018-01-03 2024-04-04 Alpine Immune Sciences, Inc. Multi-domain immunomodulatory proteins and methods of use thereof
EP3738137A1 (en) 2018-01-12 2020-11-18 The Trustees of Indiana University Electrostatic linear ion trap design for charge detection mass spectrometry
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
US20190256867A1 (en) 2018-02-01 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド F8遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
EP3768695A4 (en) 2018-02-27 2022-04-06 The Trustees of the University of Pennsylvania NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF
EP3759218A4 (en) 2018-02-28 2021-12-08 The University of North Carolina at Chapel Hill PROCEDURES AND COMPOSITIONS FOR ANTIBODY ELIMINATING VIRUS VECTORS
CA3092871A1 (en) * 2018-03-06 2019-09-12 University Of Florida Research Foundation, Incorporated Aav chimeras
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
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
CA3095911A1 (en) * 2018-04-03 2019-10-10 Cornell University Gene therapy for oxidative stress
CA3097192A1 (en) 2018-04-27 2019-10-31 Voyager Therapeutics, Inc. Methods for measuring the potency of aadc viral vectors
US12215338B2 (en) 2018-05-08 2025-02-04 Neuracle Science Co., Ltd. Adeno-associated virus (AAV) delivery of anti-FAM19A5 antibodies
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
US11821009B2 (en) 2018-05-15 2023-11-21 Cornell University Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery
US20210214749A1 (en) 2018-05-16 2021-07-15 Voyager Therapeutics, Inc. Directed evolution
US20210207167A1 (en) 2018-05-16 2021-07-08 Voyager Therapeutics, Inc. Aav serotypes for brain specific payload delivery
CA3100838A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Charge detection mass spectrometry with real time analysis and signal optimization
WO2019236143A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Apparatus and method for calibrating or resetting a charge detector
WO2019236139A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Interface for transporting ions from an atmospheric pressure environment to a low pressure environment
JP7398810B2 (ja) 2018-06-04 2023-12-15 ザ・トラスティーズ・オブ・インディアナ・ユニバーシティー 静電線形イオン・トラップにイオンを捕獲する装置および方法
EA202000353A1 (ru) 2018-06-04 2022-01-14 Калиди Биотерапьютикс, Инк. Основанные на клетках носители для потенциализации вирусной терапии
EP4391015A3 (en) 2018-06-04 2024-10-09 The Trustees of Indiana University Ion trap array for high throughput charge detection mass spectrometry
US11505782B2 (en) 2018-06-04 2022-11-22 Calidi Biotherapeutics, Inc. Cell-based vehicles for potentiation of viral therapy
AU2019287468B2 (en) 2018-06-12 2024-10-10 The University Of North Carolina At Chapel Hill Synthetic liver-tropic adeno-associated virus capsids and uses thereof
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
WO2019241758A1 (en) 2018-06-15 2019-12-19 Alpine Immune Sciences, Inc. Pd-1 variant immunomodulatory proteins and uses thereof
BR112020025268A2 (pt) 2018-06-28 2021-03-09 The University Of North Carolina At Chapel Hill Cassetes de expressão e genes cln5 otimizados e seu uso
WO2020010035A1 (en) 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Cannula system
US20210254103A1 (en) 2018-07-02 2021-08-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
CN112770812A (zh) 2018-07-24 2021-05-07 沃雅戈治疗公司 产生基因治疗制剂的系统和方法
WO2020028751A2 (en) 2018-08-03 2020-02-06 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
EP3833766A1 (en) * 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
IL280300B2 (en) 2018-08-10 2025-06-01 Univ North Carolina Chapel Hill Optimized cln7 genes and expression cassettes and their use
US20200061150A1 (en) 2018-08-23 2020-02-27 Nymox Corporation Method of inducing selective prostate glandular pharmaco-ablation with sparing of nerves and preservation of sexual function
US11242528B2 (en) 2018-08-28 2022-02-08 Actym Therapeutics, Inc. Engineered immunostimulatory bacterial strains and uses thereof
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CA3114175A1 (en) 2018-10-01 2020-04-09 The Trustees Of The University Of Pennsylvania Beta-galactosidase based aav for treating gm1 gangliosidosis
CN113166208B (zh) 2018-10-02 2024-11-22 沃雅戈治疗公司 重新定向aav衣壳的嗜性
WO2020072700A1 (en) 2018-10-02 2020-04-09 Dana-Farber Cancer Institute, Inc. Hla single allele lines
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
WO2020072844A1 (en) 2018-10-05 2020-04-09 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CN113166781A (zh) 2018-10-15 2021-07-23 沃雅戈治疗公司 在杆状病毒/Sf9系统中大规模生产rAAV的表达载体
JP2022506066A (ja) 2018-11-05 2022-01-17 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 最適化されたfig4遺伝子および発現カセットならびにそれらの使用
CA3116192C (en) 2018-11-06 2025-09-23 Calidi Biotherapeutics (Nevada), Inc. IMPROVED SYSTEMS FOR CELL-MEDIATED ONCOLYTIC VIRAL THERAPY
CN113574632B (zh) 2018-11-20 2024-07-30 印地安纳大学理事会 用于单粒子质谱分析的轨道阱
EP3884041A2 (en) 2018-11-21 2021-09-29 Indapta Therapeutics, Inc. Methods for expansion of natural killer (nk) cell subset and related compositions and methods
CA3120923A1 (en) 2018-11-26 2020-06-04 Universitat Autonoma De Barcelona Fibroblast growth factor 21 (fgf21) gene therapy
CA3120868A1 (en) 2018-11-30 2020-06-04 Alpine Immune Sciences, Inc. Cd86 variant immunomodulatory proteins and uses thereof
EP4443473A3 (en) 2018-12-03 2025-01-01 The Trustees of Indiana University Apparatus for simultaneously analyzing multiple ions with an electrostatic linear ion trap
US20220062394A1 (en) 2018-12-17 2022-03-03 The Broad Institute, Inc. Methods for identifying neoantigens
SG11202106356QA (en) 2018-12-20 2021-07-29 Vigeneron Gmbh An optimized acceptor splice site module for biological and biotechnological applications
WO2020132385A1 (en) 2018-12-21 2020-06-25 The University Of North Carolina At Chapel Hill Optimized galc genes and expression cassettes and their use
KR20240091046A (ko) 2018-12-21 2024-06-21 바이오엔테크 유에스 인크. Hla 클래스 ii-특이적 에피토프 예측 및 cd4+ t 세포 특징화를 위한 방법 및 시스템
SG11202107645RA (en) 2019-01-18 2021-08-30 Voyager Therapeutics Inc Methods and systems for producing aav particles
WO2020160508A1 (en) 2019-01-31 2020-08-06 Oregon Health & Science University Methods for using transcription-dependent directed evolution of aav capsids
CN113728108A (zh) 2019-02-15 2021-11-30 桑格摩生物治疗股份有限公司 用于生产重组aav的组合物和方法
BR112021015817A2 (pt) 2019-02-22 2021-10-13 The Trustees Of The University Of Pennsylvania Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn
SG11202108044YA (en) 2019-02-25 2021-09-29 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
US20220154211A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
EP3931337A1 (en) 2019-02-26 2022-01-05 The Trustees of The University of Pennsylvania Compositions useful in treatment of krabbe disease
US12024709B2 (en) 2019-02-27 2024-07-02 Actym Therapeutics, Inc. Immunostimulatory bacteria engineered to colonize tumors, tumor-resident immune cells, and the tumor microenvironment
JP2022524951A (ja) 2019-02-27 2022-05-11 アクティム・セラピューティクス・インコーポレイテッド 腫瘍、腫瘍常在免疫細胞および腫瘍微小環境にコロニー形成するよう操作した免疫刺激性細菌
CN113795279B (zh) 2019-03-04 2024-12-31 宾夕法尼亚州大学信托人 靶向akt通路的神经保护性基因疗法
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
JP7664848B2 (ja) 2019-04-10 2025-04-18 プリベイル セラピューティクス,インコーポレーテッド リソソーム障害のための遺伝子療法
EP3959741A1 (en) 2019-04-23 2022-03-02 The Trustees of Indiana University Identification of sample subspecies based on particle charge behavior under structural change-inducing sample conditions
EP4169535A1 (en) 2019-04-26 2023-04-26 The University of North Carolina at Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
WO2020223215A1 (en) 2019-04-29 2020-11-05 The University Of North Carolina At Chapel Hill Optimized sumf1 genes and expression cassettes and their use
WO2020223280A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
EP3962536A1 (en) 2019-04-29 2022-03-09 Voyager Therapeutics, Inc. Systems and methods for producing baculoviral infected insect cells (biics) in bioreactors
KR20220020261A (ko) 2019-05-03 2022-02-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 이염성 백질이영양증의 치료에 유용한 조성물
US20240124889A1 (en) 2019-05-07 2024-04-18 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
US11278588B2 (en) 2019-05-13 2022-03-22 Nymox Corporation Method of treating lower urinary tract symptoms with fexapotide triflutate
US11298400B2 (en) 2019-05-13 2022-04-12 Nymox Corporation Method of enhancing the therapeutic efficacy of fexapotide triflutate in treating LUTS
US12465655B2 (en) 2019-05-22 2025-11-11 The University Of North Carolina At Chapel Hill UBE3A genes and expression cassettes and their use
WO2020239995A1 (en) 2019-05-31 2020-12-03 Universitat Autònoma De Barcelona Insulin gene therapy
EP3990030A1 (en) 2019-06-27 2022-05-04 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
ES2948620T3 (es) 2019-07-10 2023-09-14 Masonic Medical Res Laboratory Vgll4 con elemento regulador CIS de UCP-1 y método de uso del mismo
EP4007814A1 (en) 2019-07-26 2022-06-08 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
US11231421B2 (en) 2019-07-31 2022-01-25 Nymox Corporation Methods of treating multifocal cancer
US11331374B2 (en) 2019-07-31 2022-05-17 Nymox Corporation Focal treatment of prostate cancer
WO2021025995A1 (en) 2019-08-02 2021-02-11 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
EP4010465A1 (en) 2019-08-09 2022-06-15 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
CA3146702A1 (en) * 2019-08-09 2021-02-18 Sergei Zolotukhin Aav capsid variants for targeting human glioblastoma cells
AU2020334147A1 (en) 2019-08-21 2022-03-31 Trames Bio, Inc. Compositions and methods for neurological diseases
EP4022070A1 (en) 2019-08-26 2022-07-06 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CN111454969B (zh) * 2019-09-02 2022-08-26 西北农林科技大学 带有双标签猪细小病毒全长感染性克隆制备方法及应用
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
WO2021061650A1 (en) 2019-09-25 2021-04-01 The Trustees Of Indiana University Apparatus and method for pulsed mode charge detection mass spectrometry
US12394502B2 (en) 2019-10-02 2025-08-19 The General Hospital Corporation Method for predicting HLA-binding peptides using protein structural features
CN114728237B (zh) 2019-10-10 2026-02-24 印地安纳大学理事会 用于识别、选择和纯化粒子的系统和方法
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ,インコーポレイテッド ニーマン・ピック病c型の治療のためのアデノ随伴ウイルスベクター
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
WO2021080975A1 (en) * 2019-10-21 2021-04-29 The Trustees Of The University Of Pennsylvania Compositions and methods for reducing cholesterol levels
AU2020401116A1 (en) 2019-12-10 2022-07-21 Takeda Pharmaceutical Company Limited Adeno associated virus vectors for the treatment of Hunter disease
EP4077684A1 (en) * 2019-12-18 2022-10-26 Genethon Production of recombinant viral vectors from plant hairy roots
JP7690209B2 (ja) 2019-12-18 2025-06-10 ザ・トラスティーズ・オブ・インディアナ・ユニバーシティー 電荷測定装置を有する質量分析計
CA3162020A1 (en) 2019-12-19 2021-06-24 James M. Wilson Compositions for treating friedreich's ataxia
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
EP4100991B1 (en) 2020-02-03 2025-01-29 The Trustees of Indiana University A charge detection mass spectrometer and related method
EP4100056A4 (en) * 2020-02-07 2024-03-06 Vecprobio, Inc. Recombinant adeno-associated viral vectors in plants
US20210284699A1 (en) * 2020-03-12 2021-09-16 California Institute Of Technology Adeno-associated viral capsids with expanded sizes
US12605465B2 (en) 2020-03-23 2026-04-21 The University Of North Carolina At Chapel Hill AAV-NAGLU vectors for treatment of mucopolysaccharidosis IIIB
WO2021202651A1 (en) 2020-04-01 2021-10-07 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
JP7713956B2 (ja) 2020-04-15 2025-07-28 ボイジャー セラピューティクス インコーポレイテッド タウ結合化合物
GB202005732D0 (en) 2020-04-20 2020-06-03 Synpromics Ltd Regulatory nucleic acid sequences
AU2021258194A1 (en) 2020-04-22 2022-11-24 Indapta Therapeutics, Inc. Natural killer (NK) cell compositions and methods for generating same
CA3177869A1 (en) 2020-05-05 2021-11-11 Duke University Cross-species compatible adeno-associated virus compositions and methods of use thereof
JP2023524577A (ja) 2020-05-05 2023-06-12 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 改変アデノ随伴ウイルス5カプシドおよびその使用
EP4146684A2 (en) 2020-05-08 2023-03-15 Alpine Immune Sciences, Inc. April and baff inhibitory immunomodulatory proteins with and without a t cell inhibitory protein and methods of use thereof
IL298128A (en) 2020-05-13 2023-01-01 Akouos Inc Compositions and methods for treating slc26a4-associated hearing loss
TWI900553B (zh) 2020-05-13 2025-10-11 美商航海家醫療公司 Aav蛋白殼之趨性重定向
KR20230017845A (ko) 2020-05-26 2023-02-06 유니버시타트 아우토노마 데 바르셀로나 중추 신경계 장애에 대한 섬유아세포 성장 인자 21(fgf21) 유전자 요법
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
CN111778219A (zh) * 2020-07-08 2020-10-16 中国农业科学院兰州兽医研究所 展示南非2型口蹄疫病毒b细胞表位猪细小病毒样颗粒的制备方法
CA3186207A1 (en) 2020-07-23 2022-01-27 Rachel BAILEY Optimized slc13a5 genes and expression cassettes and their use
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
WO2022032153A1 (en) 2020-08-06 2022-02-10 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
CA3190399A1 (en) 2020-08-24 2022-03-03 James M. Wilson Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
EP4204014A1 (en) 2020-08-26 2023-07-05 The Trustees of The University of Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
GB202013940D0 (en) 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences
AU2021358781A1 (en) 2020-10-07 2023-05-18 Tern Therapeutics, Llc Gene therapy for ocular manifestations of cln2 disease
CA3195177A1 (en) 2020-10-07 2022-04-14 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy disorders including limb girdle 2i (lgmd2i)
WO2022082109A1 (en) 2020-10-18 2022-04-21 The Trustees Of The University Of Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
WO2022093769A1 (en) 2020-10-28 2022-05-05 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
CN116490514A (zh) 2020-10-28 2023-07-25 北卡罗来纳-查佩尔山大学 用于双重聚糖结合aav2.5载体的方法和组合物
JP2023550581A (ja) 2020-10-29 2023-12-04 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 Aavカプシド及びそれを含有する組成物
AU2021375404A1 (en) 2020-11-03 2023-06-08 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
TW202237850A (zh) 2020-12-01 2022-10-01 賓州大學委員會 具有組織特異性靶向基序的新穎構成物及含有其之組成物
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
JP2024501482A (ja) 2020-12-14 2024-01-12 ビオンテック ユーエス インコーポレイテッド がん免疫療法のための組織特異的抗原
US20240052322A1 (en) 2020-12-15 2024-02-15 Pfizer Inc. HILIC UPLC-MS Method For Separating and Analyzing Intact Adeno-Associated Virus Capsid Proteins
IL303959A (en) 2020-12-23 2023-08-01 Pfizer Methods for purification of aav vectors by affinity chromatography
IL304006A (en) 2020-12-29 2023-08-01 Akouos Inc Compositions and methods for treating hearing loss and/or vision loss associated with CLRN1
WO2022147480A1 (en) 2020-12-30 2022-07-07 Ansun Biopharma, Inc. Oncolytic virus encoding sialidase and multispecific immune cell engager
TW202242124A (zh) 2021-01-14 2022-11-01 美商史崔德生物公司 靶向t細胞之aav載體
US20250345463A1 (en) 2021-01-30 2025-11-13 Universitat Autònoma De Barcelona Gene therapy for monogenic diabetes
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
AR124981A1 (es) 2021-02-26 2023-05-24 Takeda Pharmaceuticals Co Composición y métodos para el tratamiento de la enfermedad de fabry
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US11795207B2 (en) 2021-03-30 2023-10-24 AAVnerGene Inc. Modified plasma clotting factor VIII and method of use thereof
WO2022211791A1 (en) 2021-03-30 2022-10-06 AAVnerGene Inc. Modified plasma clotting factor viii and method of use thereof
US20240307553A1 (en) 2021-04-01 2024-09-19 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
IL307633A (en) 2021-04-12 2023-12-01 Univ Pennsylvania Useful preparations in the treatment of spinal muscular atrophy (SBMA)
WO2022221529A1 (en) 2021-04-16 2022-10-20 Asklepios Biopharmaceutical, Inc. Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response
US20240181084A1 (en) 2021-04-23 2024-06-06 University Of Rochester Genome Editing by Directed Non-Homologous DNA Insertion Using a Retroviral Integrase-Cas Fusion Protein and Methods of Treatment
US20250188126A1 (en) 2021-04-26 2025-06-12 Alexion Pharma International Operations Limited Adeno-associated viral vector capsids with improved tissue tropism
EP4089171A1 (en) 2021-05-12 2022-11-16 Fundación del Sector Público Estatal Centro Nacional de Investigaciones Oncológicas Carlos III (F.S.P. CNIO) Recombinant tert-encoding viral genomes and vectors
US20240252679A1 (en) 2021-05-28 2024-08-01 Shanghai Regenelead Therapies Co., Ltd. Recombinant adeno-associated virus having variant capsid, and application thereof
CN117836010A (zh) 2021-06-21 2024-04-05 优尼科生物制药有限公司 改进的裂解程序
WO2022269466A1 (en) 2021-06-22 2022-12-29 Pfizer Inc. Production of adeno-associated virus vector in insect cells
KR20240107201A (ko) 2021-08-31 2024-07-08 스카우트 바이오, 인코포레이티드 항원-결합 분자 및 이의 용도
WO2023044483A2 (en) 2021-09-20 2023-03-23 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
JP2024537775A (ja) 2021-09-30 2024-10-16 アコーオス インコーポレイテッド Kcnq4関連難聴を治療するための組成物及び方法
EP4409010A1 (en) 2021-10-02 2024-08-07 The Trustees of The University of Pennsylvania Novel aav capsids and compositions containing same
CA3234809A1 (en) 2021-10-20 2023-04-27 Steven Goldman Isolated glial progenitor cells for use in the competition treatment of age-related white matter loss
EP4426331A1 (en) 2021-11-02 2024-09-11 University of Rochester Tcf7l2 mediated remyelination in the brain
EP4426716A1 (en) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
US20250049955A1 (en) 2021-11-17 2025-02-13 Voyager Therapeutics, Inc. Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
EP4433169A1 (en) 2021-11-17 2024-09-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
TW202325850A (zh) 2021-11-29 2023-07-01 大陸商上海瑞宏迪醫藥有限公司 Aadc、gdnf多核苷酸及其用於治療帕金森病
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
WO2023111580A1 (en) 2021-12-16 2023-06-22 University Of Dundee Targeted degradation of alpha-synuclein
WO2023133574A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful for treatment of c9orf72-mediated disorders
CN119317718A (zh) 2022-01-25 2025-01-14 宾夕法尼亚州大学信托人 用于改善心脏转导和肝脏去靶向的aav衣壳
WO2023147374A2 (en) 2022-01-25 2023-08-03 Voyager Therapeutics, Inc. Baculovirus expression system
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
TW202342525A (zh) 2022-02-02 2023-11-01 美商阿科奧斯公司 用於治療前庭神經鞘瘤相關症狀之抗vegf抗體構築體及相關方法
TW202342759A (zh) 2022-02-04 2023-11-01 美商史崔德生物公司 重組腺相關病毒載體及其使用方法
CA3249551A1 (en) 2022-02-08 2023-08-17 Voyager Therapeutics, Inc. AAV Capsid Variants and Their Uses
GB202201713D0 (en) 2022-02-10 2022-03-30 Univ Dundee An affinity directed phosphatase system for targeted protein dephosphorylation
AU2023220237A1 (en) 2022-02-21 2024-08-15 Shanghai Regenelead Therapies Co., Ltd Vegf-binding molecule and pharmaceutical use thereof
CN114657152A (zh) * 2022-03-31 2022-06-24 苏州博腾生物制药有限公司 一种新型aav血清型及其制备方法
CN119301262A (zh) 2022-04-01 2025-01-10 武田药品工业株式会社 用于具有cns表现的疾病的基因疗法
EP4504797A1 (en) 2022-04-06 2025-02-12 The Trustees of The University of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
KR20250018169A (ko) 2022-04-29 2025-02-04 브로드윙 바이오 엘엘씨 이중특이적 항체 및 안구 질환의 치료 방법
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
US20250296996A1 (en) 2022-04-29 2025-09-25 Broadwing Bio, Inc. Angiopoietin-related protein 7-specific antibodies and uses thereof
EP4514844A1 (en) 2022-04-29 2025-03-05 Broadwing Bio LLC Complement factor h related 4-specific antibodies and uses thereof
WO2023220040A1 (en) * 2022-05-09 2023-11-16 Synteny Therapeutics, Inc. Erythroparvovirus with a modified capsid for gene therapy
WO2023220695A2 (en) 2022-05-13 2023-11-16 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
EP4532515A1 (en) 2022-06-02 2025-04-09 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
EP4543473A1 (en) 2022-06-22 2025-04-30 Voyager Therapeutics, Inc. Tau binding compounds
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression
CA3259902A1 (en) 2022-06-28 2024-01-04 Voyager Therapeutics, Inc. AAV Capsid Variants and Their Uses
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
US20250388626A1 (en) 2022-07-06 2025-12-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024015892A1 (en) 2022-07-13 2024-01-18 The Broad Institute, Inc. Hla-ii immunopeptidome methods and systems for antigen discovery
US20260021205A1 (en) 2022-07-15 2026-01-22 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
JP2025528068A (ja) 2022-08-03 2025-08-26 ボイジャー セラピューティクス インコーポレイテッド 血液脳関門を通過させるための組成物及び方法
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
AU2023331251A1 (en) 2022-08-25 2025-02-27 Takeda Pharmaceutical Company Limited Composition for use in the treatment of fabry disease
EP4584383A1 (en) 2022-09-07 2025-07-16 Universitat Autònoma De Barcelona Beta-hexosaminidase vectors
US20260085328A1 (en) 2022-09-08 2026-03-26 Voyager Therapeutics, Inc. Controlled expression of viral proteins
KR20250069606A (ko) 2022-09-15 2025-05-19 보이저 테라퓨틱스, 인크. 타우 결합 화합물
WO2024105638A1 (en) 2022-11-18 2024-05-23 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of hunter syndrome
WO2024124019A2 (en) 2022-12-07 2024-06-13 Ginkgo Bioworks, Inc. Aav vectors targeting hematopoietic stem cells
EP4634394A2 (en) 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
KR20250129743A (ko) 2022-12-29 2025-08-29 보이저 테라퓨틱스, 인크. Mapt를 조절하기 위한 조성물 및 방법
EP4658294A2 (en) 2023-02-02 2025-12-10 University of Rochester Competitive replacement of glial cells
AU2023427408A1 (en) 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
EP4698637A1 (en) * 2023-04-18 2026-02-25 Regents of the University of Minnesota Capsid engineered adeno-associated viruses
WO2024226790A1 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024227154A1 (en) 2023-04-28 2024-10-31 Broadwing Bio Llc Complement component 3 (c3)-specific antibodies and uses thereof
AU2024266140A1 (en) 2023-05-02 2025-09-11 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2024229164A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
KR20260021096A (ko) 2023-05-03 2026-02-12 보이저 테라퓨틱스, 인크. Cdkl5 결핍과 관련된 장애의 치료를 위한 조성물 및 방법
EP4705487A2 (en) 2023-05-03 2026-03-11 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to frataxin deficiency
WO2024229173A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
AU2024266519A1 (en) 2023-05-03 2025-12-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to syntaxin-binding protein 1 deficiency
TW202444917A (zh) 2023-05-04 2024-11-16 美商航海家醫療公司 Aav衣殼變異體及其用途
KR20260010739A (ko) 2023-05-11 2026-01-21 유니버시티 하스피탈즈 클리블랜드 메디컬 센터 항불안 치료
US12531162B1 (en) * 2023-05-31 2026-01-20 Northeastern University Multi-dimensional phenotypic space for genotype to phenotype mapping and intelligent design of cancer drug therapies using a deep learning net
TW202516019A (zh) 2023-06-29 2025-04-16 賓州大學委員會 具中樞神經系統靶向模體的突變aav及含有其之組成物
IL326390A (en) 2023-08-10 2026-04-01 Univ Pennsylvania Compositions and methods for treating spinal muscular atrophy
WO2025038430A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025038796A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2025038802A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
WO2025038805A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
TW202523846A (zh) 2023-08-16 2025-06-16 美商航海家醫療公司 用於治療與共濟蛋白(frataxin)不足有關之疾病之組合物及方法
WO2025038795A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
TW202508610A (zh) 2023-08-23 2025-03-01 大陸商上海瑞宏迪醫藥有限公司 醫藥組成物及其用途
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
WO2025097055A2 (en) 2023-11-02 2025-05-08 The Broad Institute, Inc. Compositions and methods of use of t cells in immunotherapy
US12562256B2 (en) * 2023-11-07 2026-02-24 New York University Systems, methods and computer-accessible medium for identifying target pairs for CAR-T therapy
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
WO2025122536A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025122532A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
WO2025122548A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2025122530A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025122543A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to syntaxin-binding protein 1 deficiency
WO2025122644A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2025122634A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
TW202540436A (zh) 2023-12-05 2025-10-16 美商航海家醫療公司 用於治療與共濟蛋白(frataxin)缺陷相關之病症之組合物及方法
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
WO2025137219A1 (en) 2023-12-21 2025-06-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025147436A1 (en) 2024-01-03 2025-07-10 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
TW202548022A (zh) 2024-03-03 2025-12-16 美商帕西奇生物公司 用於治療神經退化性病症之重組腺相關病毒
WO2025235734A2 (en) 2024-05-09 2025-11-13 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025250454A1 (en) 2024-05-28 2025-12-04 University Of Rochester Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo
WO2026010722A1 (en) 2024-07-03 2026-01-08 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2026015039A1 (en) 2024-07-09 2026-01-15 Bial - Portela & Ca., S.A. Gene therapy for treatment of syngap1-related epileptic encephalopathy
WO2026017965A1 (en) 2024-07-19 2026-01-22 The University Court Of The University Of Edinburgh Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions
WO2026055342A1 (en) 2024-09-04 2026-03-12 Arsenal Biosciences, Inc. Synthetic pathway activators

Family Cites Families (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
AU7966987A (en) 1986-09-08 1988-04-07 Applied Biotechnology, Inc. Empty viral capsid vaccines
US5916563A (en) 1988-11-14 1999-06-29 United States Of America Parvovirus protein presenting capsids
US6001371A (en) 1988-11-14 1999-12-14 The United States Of America As Represented By The Department Of Health And Human Services Parvovirus capsids
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
ES2026826A6 (es) 1991-03-26 1992-05-01 Ercros Sa Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados.
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
EP0693940B1 (en) 1993-04-06 2002-11-13 Targeted Genetics Corporation Hybrid genes for use in the production of t h?-independent cytotoxic t cells
US5834441A (en) 1993-09-13 1998-11-10 Rhone-Poulenc Rorer Pharmaceuticals Inc. Adeno-associated viral (AAV) liposomes and methods related thereto
FR2716893B1 (fr) * 1994-03-03 1996-04-12 Rhone Poulenc Rorer Sa Virus recombinants, leur préparation et leur utilisation thérapeutique.
ATE386131T1 (de) 1994-04-13 2008-03-15 Univ Rockefeller Aav-vermittelte überbringung von dna in zellen des nervensystems
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US5872005A (en) 1994-11-03 1999-02-16 Cell Genesys Inc. Packaging cell lines for adeno-associated viral vectors
US5843742A (en) 1994-12-16 1998-12-01 Avigen Incorporated Adeno-associated derived vector systems for gene delivery and integration into target cells
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US5773289A (en) 1995-06-06 1998-06-30 University Of Pittsburgh AAV directed targeted integration
CA2224907A1 (en) 1995-07-25 1997-02-13 Introgene B.V. Methods and means for targeted gene delivery
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US5945335A (en) 1995-11-09 1999-08-31 Avigen, Inc. Adenovirus helper-free system for producing recombinant AAV virions lacking oncogenic sequences
US5874304A (en) 1996-01-18 1999-02-23 University Of Florida Research Foundation, Inc. Humanized green fluorescent protein genes and methods
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5846528A (en) 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US5741492A (en) 1996-01-23 1998-04-21 St. Jude Children's Research Hospital Preparation and use of viral vectors for mixed envelope protein vaccines against human immunodeficiency viruses
US5952221A (en) 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
US5722177A (en) * 1996-03-20 1998-03-03 Reilly, Iii; Earl F. Golf club putter fitting apparatus and method
US5638326A (en) 1996-04-05 1997-06-10 Advanced Micro Devices, Inc. Parallel page buffer verify or read of cells on a word line using a signal from a reference cell in a flash memory device
EP0927044A4 (en) 1996-04-16 1999-09-08 Immusol Inc DEFINED TARGET VIRAL VECTORS
US5780447A (en) 1996-06-14 1998-07-14 St. Jude Children's Research Hospital Recombinant adeno-associated viral vectors
US5866552A (en) 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
WO1998009524A1 (en) 1996-09-06 1998-03-12 Chiron Corporation Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors
WO1998011244A2 (en) * 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services Aav4 vector and uses thereof
US5922315A (en) * 1997-01-24 1999-07-13 Genetic Therapy, Inc. Adenoviruses having altered hexon proteins
EP1005376B1 (en) 1997-03-14 2010-11-03 The Children's Hospital of Philadelphia Compositions for use in gene therapy for treatment of hemophilia
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US5962274A (en) 1998-03-13 1999-10-05 Wake Forest University Viral vector directed to predetermined target cells
US6797505B2 (en) * 1998-05-27 2004-09-28 Cell Genesys, Inc. Recombinant AAV vectors for gene therapy of hemophilia A
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
US6984517B1 (en) * 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
DE19827457C1 (de) 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
ATE362542T1 (de) 1998-11-05 2007-06-15 Univ Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
AU780231B2 (en) * 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
DE19933288A1 (de) * 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
DE19933719A1 (de) 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
YANG ET AL. (1998) HWM GENE THER 9:1929-1937 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2001255575B2 (en) * 2000-04-28 2006-08-31 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids

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