LT3250239T - Kapsidė - Google Patents

Kapsidė

Info

Publication number
LT3250239T
LT3250239T LTEP16721917.9T LT16721917T LT3250239T LT 3250239 T LT3250239 T LT 3250239T LT 16721917 T LT16721917 T LT 16721917T LT 3250239 T LT3250239 T LT 3250239T
Authority
LT
Lithuania
Prior art keywords
crossed
Prior art date
Application number
LTEP16721917.9T
Other languages
English (en)
Lithuanian (lt)
Inventor
Amit Nathwani
Allison DANE
Original Assignee
Ucl Business Plc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ucl Business Plc filed Critical Ucl Business Plc
Publication of LT3250239T publication Critical patent/LT3250239T/lt

Links

Classifications

    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (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)
LTEP16721917.9T 2015-05-11 2016-05-10 Kapsidė LT3250239T (lt)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
LT3250239T true LT3250239T (lt) 2019-03-25

Family

ID=53489438

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEP16721917.9T LT3250239T (lt) 2015-05-11 2016-05-10 Kapsidė

Country Status (18)

Country Link
US (3) US10414803B2 (enExample)
EP (2) EP3250239B1 (enExample)
JP (2) JP6619454B2 (enExample)
KR (1) KR20180019543A (enExample)
CN (1) CN107864653B (enExample)
AU (1) AU2016261454B2 (enExample)
CA (1) CA2985945C (enExample)
DK (1) DK3250239T3 (enExample)
ES (1) ES2711349T3 (enExample)
GB (1) GB201508026D0 (enExample)
HR (1) HRP20190442T1 (enExample)
HU (1) HUE042693T2 (enExample)
LT (1) LT3250239T (enExample)
PL (1) PL3250239T3 (enExample)
PT (1) PT3250239T (enExample)
TR (1) TR201901582T4 (enExample)
TW (1) TW201704253A (enExample)
WO (1) WO2016181123A1 (enExample)

Families Citing this family (47)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
GB201508026D0 (en) * 2015-05-11 2015-06-24 Ucl Business Plc Capsid
US10745447B2 (en) 2015-09-28 2020-08-18 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
GB201608046D0 (en) 2016-05-09 2016-06-22 Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The Treatment of complement-mediated disorders
EP4361277A3 (en) 2016-12-30 2024-07-31 The Trustees of The University of Pennsylvania Gene therapy for treating wilson's disease
EP3645021A4 (en) * 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
WO2019191701A1 (en) * 2018-03-30 2019-10-03 The Board Of Trustees Of Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
SG11202009451VA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
MX2020010464A (es) * 2018-04-03 2021-01-29 Vectores de virus que evitan anticuerpos.
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
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
PT3906066T (pt) 2019-01-04 2024-02-06 Ultragenyx Pharmaceutical Inc Constructos de terapia génica para o tratamento da doença de wilson
SG11202110146VA (en) 2019-03-21 2021-10-28 Stridebio Inc Recombinant adeno-associated virus vectors
EP3715358A1 (en) * 2019-03-28 2020-09-30 Universität zu Köln Mutated adeno-associated virus capsid proteins, aav particle comprising the same and liver directed aav vector gene therapy
LT3722434T (lt) 2019-04-12 2022-11-10 Freeline Therapeutics Limited Plazmidžių sistema
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
AU2020330121B2 (en) * 2019-08-14 2025-10-30 University Of Florida Research Foundation, Incorporated AAV capsid variants for gene therapy
CN115087738A (zh) * 2019-08-29 2022-09-20 小利兰·斯坦福大学托管委员会 用于穿越人血脑屏障的腺相关病毒载体
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
AU2021228368A1 (en) * 2020-02-25 2022-10-20 Children's Medical Research Institute Adeno-associated virus capsid polypeptides and vectors
CN116209768A (zh) * 2020-07-03 2023-06-02 吉尼松公司 用于通过高变区交换工程化新杂合aav衣壳的方法
CN116121274B (zh) * 2020-07-29 2024-11-19 北京三诺佳邑生物技术有限责任公司 一组肝靶向新型腺相关病毒的获得及其应用
TW202227632A (zh) 2020-08-19 2022-07-16 美商史崔德生物公司 用於治療雷特症候群之腺相關病毒載體
WO2022060916A1 (en) 2020-09-15 2022-03-24 Regenxbio Inc. Vectorized antibodies for anti-viral therapy
US20250326861A1 (en) 2020-09-15 2025-10-23 Regenxbio Inc. Vectorized lanadelumab and administration thereof
GB202016254D0 (en) 2020-10-13 2020-11-25 Freeline Therapeutics Ltd Plasmid system
EP4236999A1 (en) 2020-10-28 2023-09-06 RegenxBio Inc. Vectorized anti-tnf-alfa antibodies for ocular indications
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
WO2022094255A2 (en) 2020-10-29 2022-05-05 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
CN113121654B (zh) * 2021-04-19 2023-11-17 信念医药科技(上海)有限公司 腺相关病毒衣壳蛋白及包含其的腺相关病毒载体
WO2022226301A1 (en) * 2021-04-23 2022-10-27 The University Of North Carolina At Chapel Hill Chimeric heart tropic aav capsids
GB202109231D0 (en) 2021-06-25 2021-08-11 Freeline Therapeutics Ltd Promoter
CN118201947B (zh) 2021-10-28 2025-03-21 上海天泽云泰生物医药有限公司 腺相关病毒衣壳
GB202205514D0 (en) 2022-04-13 2022-05-25 Freeline Therapeutics Ltd Mechanical lysis
AR129215A1 (es) 2022-05-03 2024-07-31 Regenxbio Inc Anticuerpos anti-complemento y agentes del complemento vectorizados y administración de estos
EP4518973A1 (en) 2022-05-03 2025-03-12 RegenxBio Inc. Vectorized anti-tnf-alpha inhibitors for ocular indications
GB202214328D0 (en) 2022-09-29 2022-11-16 Freeline Therapeutics Ltd Method
WO2024238867A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof
WO2025087695A1 (en) 2023-10-23 2025-05-01 Ascend Advanced Therapies Ltd Method for producing recombinant adeno-associated virus particles
EP4628576A1 (en) 2024-04-07 2025-10-08 Ascend Advanced Therapies GmbH Microrna molecules for use in methods for improving recombinant aav production
WO2025217230A1 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
WO2025238067A1 (en) 2024-05-15 2025-11-20 Ascend Advanced Therapies Gmbh Novel vector system for aav production

Family Cites Families (101)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
WO1999023107A1 (en) 1997-10-31 1999-05-14 Maxygen, Incorporated Modification of virus tropism and host range by viral genome shuffling
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
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
EP1048726B1 (en) 1999-04-27 2006-07-26 Négrier, Claude Modified factor VIII cDNA
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
US20020045264A1 (en) 2000-03-14 2002-04-18 During Matthew J. Production of chimeric capsid vectors
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
KR101014207B1 (ko) 2001-11-13 2011-02-14 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 아데노-결합 바이러스 (aav) 서열을 검출 및/또는 확인하는 방법 및 그 방법에 의해 확인된 신규한 서열을 분리하는 방법
CA2469785C (en) 2001-12-17 2014-04-22 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
WO2003092594A2 (en) 2002-04-30 2003-11-13 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
PT2657247T (pt) 2003-06-19 2017-06-29 Genzyme Corp Viriões aav com imunorreatividade diminuída e seus usos
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
EP2298926A1 (en) 2003-09-30 2011-03-23 The Trustees of The University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
WO2005072364A2 (en) 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
EP1893754A4 (en) 2005-05-31 2009-01-21 Neurologix Inc NEW GLUTAMIC ACID DECARBOXYLASE (GAD) CHIMESES AND USE PROCESSES
WO2007089632A2 (en) 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
JP5349306B2 (ja) 2006-08-21 2013-11-20 ジェネンテック, インコーポレイテッド アザベンゾチオフェニル化合物および使用方法
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
EP3492596A1 (en) 2007-04-09 2019-06-05 University of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
ES2602610T3 (es) * 2007-05-31 2017-02-21 Medigene Ag Proteína estructural mutada de un 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
US9464119B2 (en) 2009-03-04 2016-10-11 Deutsches Krebsforschungszentrum Assembly activating protein (AAP) and its use for the manufacture of parvovirus particles essentially consisting of VP3
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
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
US10385112B2 (en) 2013-03-14 2019-08-20 Asklepios Biopharmaceutical, Inc. Modified soluble VEGF receptor-1 genes and vectors for gene therapy
CN105163764B (zh) 2013-03-15 2019-11-12 北卡罗来纳-查佩尔山大学 双重聚糖结合aav载体的方法和组合物
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
MX2016001044A (es) 2013-07-22 2016-09-09 The Children's Hospital Of Philadelphia Aav variante y composiciones, metodos y usos para transferencia genica a celulas, organos y tejidos.
LT3039146T (lt) 2013-08-27 2020-07-27 Research Institute at Nationwide Children`s Hospital Produktai ir būdai, skirti amiotropinės lateralinės sklerozės gydymui
CN115141260B (zh) * 2013-10-11 2025-05-20 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
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
CA2941640A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
FI3851537T3 (fi) 2014-04-25 2024-03-21 Genethon Hyperbilirubinemian hoito
JP6741591B2 (ja) 2014-05-02 2020-08-19 ジェンザイム・コーポレーション 網膜およびcns遺伝子治療用aavベクター
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
AU2015335923B2 (en) 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
AU2015349759B2 (en) 2014-11-21 2022-01-06 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system
GB201502137D0 (en) 2015-02-09 2015-03-25 Ucl Business Plc Treatment
WO2016133917A1 (en) 2015-02-16 2016-08-25 University Of Florida Research Foundation Raav vector compositions, methods for targeting vascular endothelial cells and use in treatment of type i diabetes
EP3262162A4 (en) 2015-02-23 2018-08-08 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
CN114480502A (zh) 2015-03-02 2022-05-13 阿德夫拉姆生物技术股份有限公司 用于将多核苷酸玻璃体内递送到视网膜视锥的组合物和方法
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
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
CA3019315A1 (en) 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
GB201508026D0 (en) * 2015-05-11 2015-06-24 Ucl Business Plc Capsid
CN108138159A (zh) 2015-06-23 2018-06-08 费城儿童医院 经修饰的因子ix、以及用于基因转移到细胞、器官和组织的组合物、方法和用途
WO2017015102A1 (en) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
WO2017023724A1 (en) 2015-07-31 2017-02-09 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
US10745447B2 (en) 2015-09-28 2020-08-18 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
WO2017066764A2 (en) 2015-10-16 2017-04-20 William Marsh Rice University Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
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
US10548947B2 (en) 2015-11-05 2020-02-04 Bamboo Therapeutics, Inc. Modified Friedreich ataxia genes and vectors for gene therapy
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
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
SG11201804713VA (en) 2015-12-11 2018-07-30 California Inst Of Techn TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
EP4659816A3 (en) 2015-12-14 2025-12-31 The University of North Carolina at Chapel Hill MODIFIED CAPSID PROTEINS FOR IMPROVED PARVOVIRUS VECTOR DELIVERY
US10179176B2 (en) 2016-02-16 2019-01-15 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
EP3429605A4 (en) 2016-03-18 2019-09-18 The Children's Hospital of Philadelphia THERAPEUTIC FOR THE 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
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
CN117904112A (zh) 2016-05-18 2024-04-19 沃雅戈治疗公司 调节性多核苷酸
CN109843913B (zh) 2016-06-16 2024-03-08 柏林夏洛特医科大学 神经肽表达载体以及用于治疗癫痫的方法
IL305149A (en) 2016-07-26 2023-10-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
KR20240172773A (ko) 2016-08-15 2024-12-10 젠자임 코포레이션 Aav의 검출 방법
WO2018045347A1 (en) 2016-09-02 2018-03-08 Spark Therapeutics, Inc. Methods and vectors for treating cns disorders
EP3293259A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
CA3035868A1 (en) 2016-09-12 2018-03-15 Genethon Acid-alpha glucosidase variants and uses thereof
EP3293203A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
WO2018081476A2 (en) 2016-10-27 2018-05-03 Intima Bioscience, Inc. Viral methods of t cell therapy
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
EP3592848A1 (en) 2017-03-10 2020-01-15 Genethon Treatment of glycogen storage disease iii
CN117801075A (zh) 2017-03-15 2024-04-02 北卡罗来纳-查佩尔山大学 多倍体腺相关病毒载体及其制备和使用方法
EP4219806A3 (en) 2017-04-11 2023-09-27 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
CN119491003A (zh) 2017-05-05 2025-02-21 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
MY207281A (en) 2017-06-07 2025-02-13 Spark Therapeutics Inc Enhancing agents for improved cell transfection and/or raav vector production
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
AU2018291023B2 (en) 2017-06-30 2023-11-02 Spark Therapeutics, Inc. AAV vector column purification methods
SG11202000840YA (en) 2017-07-31 2020-02-27 Reflection Biotechnologies Ltd Cellular models of and therapies for ocular diseases
KR20240160648A (ko) 2017-08-01 2024-11-11 스파크 테라퓨틱스, 인코포레이티드 인자 viii (fviii) 유전자 요법 방법
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
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

Also Published As

Publication number Publication date
HRP20190442T1 (hr) 2019-05-03
CA2985945A1 (en) 2016-11-17
DK3250239T3 (en) 2019-03-11
PT3250239T (pt) 2019-02-19
CN107864653B (zh) 2022-06-21
EP3250239A1 (en) 2017-12-06
US10414803B2 (en) 2019-09-17
AU2016261454B2 (en) 2019-07-11
HUE042693T2 (hu) 2019-07-29
TR201901582T4 (tr) 2019-02-21
JP2020096587A (ja) 2020-06-25
PL3250239T3 (pl) 2019-05-31
KR20180019543A (ko) 2018-02-26
GB201508026D0 (en) 2015-06-24
US20250127922A1 (en) 2025-04-24
JP2018520997A (ja) 2018-08-02
WO2016181123A1 (en) 2016-11-17
JP6619454B2 (ja) 2019-12-11
EP3511021A1 (en) 2019-07-17
US20180111965A1 (en) 2018-04-26
US12138318B2 (en) 2024-11-12
AU2016261454A1 (en) 2017-11-23
TW201704253A (zh) 2017-02-01
EP3250239B1 (en) 2018-12-12
CA2985945C (en) 2022-09-06
CN107864653A (zh) 2018-03-30
ES2711349T3 (es) 2019-05-03
US20200079821A1 (en) 2020-03-12

Similar Documents

Publication Publication Date Title
LT3250239T (lt) Kapsidė
DK3331869T3 (da) Muscarinagonister
DK3277719T3 (da) Polypeptider
EP3316375A4 (en) Redox flow cell
DK3305788T3 (da) Janus-kinase-hæmmer
DK3360890T3 (da) Genterapi
DK3310450T3 (da) Olie-vand-separator
EP3290364A4 (en) Conveyor belt wear monitoring system
DK3270930T3 (da) Præeklampsi
EP3381654A4 (en) Liquid blow molding method
EP3393959A4 (en) GRAB
DK3394281T3 (da) Gærcelle
EP3363322A4 (en) Hair extender
DE112015006486A5 (de) Inkubationsrinne
EP3390373C0 (en) DOTA SUMMARY
EP3548009A4 (en) POLYMERIC NANOPARTICLES
DK3274482T3 (da) Stenborsknop
EP3377179A4 (en) DEUTERED EPI-743
DE102015206660A8 (de) Plattenaufteilanlage
DE112015005906A5 (de) Gurtschlossbringer
DE112016005599A5 (de) Strahlungsgrill
EP3347241A4 (en) Protective buffer strap
EP3307242A4 (en) PICn-01
EP3308871A4 (en) CRIMPING DEVICE
ES1140083Y (es) Descompatactador tetrafunción