[go: up one dir, main page]

HUE042693T2 - Kapszid - Google Patents

Kapszid

Info

Publication number
HUE042693T2
HUE042693T2 HUE16721917A HUE16721917A HUE042693T2 HU E042693 T2 HUE042693 T2 HU E042693T2 HU E16721917 A HUE16721917 A HU E16721917A HU E16721917 A HUE16721917 A HU E16721917A HU E042693 T2 HUE042693 T2 HU E042693T2
Authority
HU
Hungary
Prior art keywords
capsid
Prior art date
Application number
HUE16721917A
Other languages
English (en)
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 HUE042693T2 publication Critical patent/HUE042693T2/hu

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)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
HUE16721917A 2015-05-11 2016-05-10 Kapszid HUE042693T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
GBGB1508026.0A GB201508026D0 (en) 2015-05-11 2015-05-11 Capsid

Publications (1)

Publication Number Publication Date
HUE042693T2 true HUE042693T2 (hu) 2019-07-29

Family

ID=53489438

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE16721917A HUE042693T2 (hu) 2015-05-11 2016-05-10 Kapszid

Country Status (18)

Country Link
US (3) US10414803B2 (hu)
EP (2) EP3511021A1 (hu)
JP (2) JP6619454B2 (hu)
KR (1) KR20180019543A (hu)
CN (1) CN107864653B (hu)
AU (1) AU2016261454B2 (hu)
CA (1) CA2985945C (hu)
DK (1) DK3250239T3 (hu)
ES (1) ES2711349T3 (hu)
GB (1) GB201508026D0 (hu)
HR (1) HRP20190442T1 (hu)
HU (1) HUE042693T2 (hu)
LT (1) LT3250239T (hu)
PL (1) PL3250239T3 (hu)
PT (1) PT3250239T (hu)
TR (1) TR201901582T4 (hu)
TW (1) TW201704253A (hu)
WO (1) WO2016181123A1 (hu)

Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
GB201508026D0 (en) * 2015-05-11 2015-06-24 Ucl Business Plc Capsid
JP7064214B2 (ja) 2015-09-28 2022-05-10 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 抗体回避性ウイルスベクターのための方法および組成物
GB201608046D0 (en) 2016-05-09 2016-06-22 Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The Treatment of complement-mediated disorders
EP3645021A4 (en) * 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
US11608510B2 (en) * 2018-03-30 2023-03-21 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
SG11202009450SA (en) 2018-04-03 2020-10-29 Stridebio Inc Virus vectors for targeting ophthalmic tissues
BR112020020195A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
KR20210006357A (ko) 2018-04-03 2021-01-18 스트라이드바이오 인코포레이티드 항체-회피 바이러스 벡터
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
EP4269579A3 (en) 2019-01-04 2024-01-24 Ultragenyx Pharmaceutical Inc. Gene therapy constructs for treating wilson disease
MX2021011468A (es) 2019-03-21 2021-12-15 Vectores de virus adenoasociados recombinantes.
EP3715358A1 (en) * 2019-03-28 2020-09-30 Universität zu Köln Mutated adeno-associated virus capsid proteins, aav particle comprising the same and liver directed aav vector gene therapy
PT3722434T (pt) 2019-04-12 2022-10-18 Freeline Therapeutics Ltd Sistema plasmídico
US20220162642A1 (en) 2019-04-12 2022-05-26 Freeline Therapeutics Limited Plasmid system
WO2021009805A1 (ja) * 2019-07-12 2021-01-21 株式会社遺伝子治療研究所 ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2021030764A1 (en) * 2019-08-14 2021-02-18 University Of Florida Research Foundation, Incorporated Aav capsid variants for gene therapy
EP4022073A4 (en) * 2019-08-29 2023-12-20 The Board Of Trustees Of The Leland Stanford Junior University ADENO-ASSOCIATED VIRAL VECTORS TO CROSS THE HUMAN BLOOD-BRAIN BARRIER
US11905523B2 (en) 2019-10-17 2024-02-20 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick Disease type-C
US20230093183A1 (en) * 2020-02-25 2023-03-23 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
CN111876432B (zh) * 2020-07-29 2022-10-28 舒泰神(北京)生物制药股份有限公司 一组肝靶向新型腺相关病毒的获得及其应用
CN116745409A (zh) 2020-08-19 2023-09-12 萨雷普塔治疗公司 用于治疗雷特综合征的腺相关病毒载体
EP4213890A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized lanadelumab and administration thereof
WO2022060916A1 (en) 2020-09-15 2022-03-24 Regenxbio Inc. Vectorized antibodies for anti-viral therapy
GB202016254D0 (en) 2020-10-13 2020-11-25 Freeline Therapeutics Ltd Plasmid system
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
CN116528892A (zh) 2020-10-28 2023-08-01 再生生物股份有限公司 用于眼部适应症的载体化抗TNF-α抗体
US20230390418A1 (en) 2020-10-29 2023-12-07 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
CN113121654B (zh) * 2021-04-19 2023-11-17 信念医药科技(上海)有限公司 腺相关病毒衣壳蛋白及包含其的腺相关病毒载体
WO2022226301A1 (en) * 2021-04-23 2022-10-27 The University Of North Carolina At Chapel Hill Chimeric heart tropic aav capsids
GB202109231D0 (en) 2021-06-25 2021-08-11 Freeline Therapeutics Ltd Promoter
WO2023072181A1 (en) * 2021-10-28 2023-05-04 Shanghai Vitalgen Biopharma Co., Ltd. Adeno-associated virus capsid
GB202205514D0 (en) 2022-04-13 2022-05-25 Freeline Therapeutics Ltd Mechanical lysis
WO2023215807A1 (en) 2022-05-03 2023-11-09 Regenxbio Inc. VECTORIZED ANTI-TNF-α INHIBITORS FOR OCULAR INDICATIONS
AR129215A1 (es) 2022-05-03 2024-07-31 Regenxbio Inc Anticuerpos anti-complemento y agentes del complemento vectorizados y administración de estos
GB202214328D0 (en) 2022-09-29 2022-11-16 Freeline Therapeutics Ltd Method
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof
WO2024238867A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
WO2025087695A1 (en) 2023-10-23 2025-05-01 Ascend Advanced Therapies Ltd Method for producing recombinant adeno-associated virus particles

Family Cites Families (101)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6156303A (en) * 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
AU1449499A (en) 1997-10-31 1999-05-24 Maxygen, Incorporated Modification of virus tropism and host range by viral genome shuffling
DE69941905D1 (de) 1998-11-10 2010-02-25 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
EP1026250A1 (en) 1998-12-02 2000-08-09 Aventis Behring GmbH DNA-constructs of blood clotting factors and P-selectin
EP1010762A1 (en) 1998-12-02 2000-06-21 Aventis Behring Gesellschaft mit beschränkter Haftung DNA constructs of blood clotting factors and P-Selectin
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
JP2003526377A (ja) 2000-03-14 2003-09-09 ニユーロロジクス・インコーポレーテツド キメラキャプシドベクターの製造
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
EP1453547B1 (en) 2001-12-17 2016-09-21 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
AU2003223775A1 (en) 2002-04-30 2003-11-17 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
DK2357189T3 (en) 2003-06-19 2017-06-19 Genzyme Corp AAV virions with reduced immunoreactivity and applications thereof
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
CA2537793C (en) 2003-09-30 2013-07-23 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
CN101124328A (zh) 2004-12-15 2008-02-13 北卡罗来纳查佩尔山大学 嵌合载体
WO2006130639A2 (en) 2005-05-31 2006-12-07 Neurologix, Inc. Novel glutamic acid decarboxylase (gad) chimera and methods of use
WO2007089632A2 (en) 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
CA2660963A1 (en) 2006-08-21 2008-02-28 Genentech, Inc. Aza-benzothiophenyl compounds and methods of use
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
HRP20161150T1 (hr) 2007-04-09 2016-11-18 University Of Florida Research Foundation, Inc. PRIPRAVCI rAAV VEKTORA KOJI IMAJU TIROZIN-MODIFICIRANE CAPSID PROTEINE I POSTUPCI ZA UPORABU
DK2158211T3 (en) * 2007-05-31 2016-12-05 Medigene Ag Mutated structural protein of a parvovirus
WO2009137006A2 (en) 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
ES2634118T3 (es) 2009-02-11 2017-09-26 The University Of North Carolina At Chapel Hill Vectores de virus modificados y métodos para fabricar y utilizar los mismos
PL2403867T3 (pl) 2009-03-04 2019-12-31 Deutsches Krebsforschungszentrum Białko aktywujące składanie (aap) i jego zastosowanie do wytwarzania cząstek parwowirusa zasadniczo składających się z vp3
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP3147295B2 (en) 2011-08-24 2023-11-22 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
WO2013063379A1 (en) 2011-10-28 2013-05-02 University Of North Carolina At Chapel Hill Cell line for production of adeno-associated virus
EP3738974A1 (en) 2012-09-28 2020-11-18 The University of North Carolina at Chapel Hill Aav vectors targeted to oligodendrocytes
WO2014064277A1 (en) 2012-10-26 2014-05-01 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
ES2667209T3 (es) 2013-03-14 2018-05-10 Asklepios Biopharmaceutical, Inc. Genes del receptor-1 de VEGF soluble modificado y vectores para terapia génica
PT2968605T (pt) 2013-03-15 2022-09-22 Univ North Carolina Chapel Hill Métodos e composições para vetores aav de ligação dupla de glicano
CA2917018A1 (en) 2013-05-21 2014-12-04 University Of Florida Research Foundation, Inc. Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer
KR20220041963A (ko) 2013-07-22 2022-04-01 더 칠드런스 호스피탈 오브 필라델피아 변종 aav 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
HUE061629T2 (hu) 2013-08-27 2023-07-28 Res Inst Nationwide Childrens Hospital Termékek és módszerek amyotrophiás lateralis sclerosis kezelésére
NZ718926A (en) * 2013-10-11 2021-12-24 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
US20170007720A1 (en) 2014-02-21 2017-01-12 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
EP3134530B1 (en) 2014-04-25 2019-06-12 Genethon Treatment of hyperbilirubinemia
AU2015252797C1 (en) 2014-05-02 2021-04-22 Genzyme Corporation AAV vectors for retinal and CNS gene therapy
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
EP3221453A4 (en) 2014-11-21 2018-09-12 The University of North Carolina at Chapel Hill Aav vectors targeted to the central nervous system
GB201502137D0 (en) 2015-02-09 2015-03-25 Ucl Business Plc Treatment
US10648000B2 (en) 2015-02-16 2020-05-12 University Of Florida Research Foundation, Incorporated rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes
EP3262162A4 (en) 2015-02-23 2018-08-08 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
BR112017018846A2 (pt) 2015-03-02 2018-07-31 Adverum Biotechnologies, Inc. composições e métodos para entrega intravítrea de polinucleotídeos a cones retinianos.
MX388331B (es) 2015-03-24 2025-03-19 Univ California Variantes de virus adenoasociado y métodos de uso de estas
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
JP6831779B2 (ja) 2015-06-23 2021-02-17 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia 修飾された第ix因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用
WO2017015102A1 (en) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
US20190000940A1 (en) 2015-07-31 2019-01-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
JP7064214B2 (ja) 2015-09-28 2022-05-10 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 抗体回避性ウイルスベクターのための方法および組成物
WO2017066764A2 (en) 2015-10-16 2017-04-20 William Marsh Rice University Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
HK1258413A1 (zh) 2015-10-29 2019-11-08 Voyager Therapeutics, Inc. 遞送中樞神經系統(cns)靶向多核苷酸
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
CN108348621A (zh) 2015-11-05 2018-07-31 竹治疗股份有限公司 用于基因治疗的经修饰的弗里德赖希共济失调基因及载体
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
JP7066619B2 (ja) 2015-12-11 2022-05-13 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス(aav)を誘導するための標的指向性ペプチド
RU2751953C2 (ru) 2015-12-14 2021-07-21 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Модифицированные капсидные белки для улучшения доставки парвовирусных векторов
AU2017219865B2 (en) 2016-02-16 2023-04-13 The Board Of Trustees Of The Leland Stanford Junior University Novel 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
WO2017180896A1 (en) 2016-04-14 2017-10-19 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
CA3024448A1 (en) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Modulatory polynucleotides
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
EP3491008A2 (en) 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
DK3497207T3 (da) 2016-08-15 2021-03-22 Genzyme Corp Fremgangsmåder til påvisning af aav
MX2019002518A (es) 2016-09-02 2019-07-18 Spark Therapeutics Inc Metodos y vectores para el tratamiento de trastornos del snc.
KR20240001721A (ko) 2016-09-12 2024-01-03 제네똥 산-알파 글루코시다제 변이체 및 이의 용도
EP3293203A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
EP3293259A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
WO2018071831A1 (en) 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
AU2017347854B2 (en) 2016-10-27 2022-12-08 Intima Bioscience, Inc. Viral methods of T cell therapy
EA038695B1 (ru) 2017-02-15 2021-10-06 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Способы и композиции для переноса генов по сосудистой сети
US11541131B2 (en) 2017-03-10 2023-01-03 Genethon Treatment of glycogen storage disease III
AU2018234695B2 (en) 2017-03-15 2024-10-03 The University Of North Carolina At Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
US20200370039A1 (en) 2017-04-11 2020-11-26 Ruprecht-Karls-Universitat 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
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 筋萎縮性側索硬化症(als)治療組成物および方法
US11752181B2 (en) 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
EP3635108A4 (en) 2017-06-07 2021-03-31 Spark Therapeutics, Inc. ACTIVATION AGENTS INTENDED FOR ENHANCED CELL TRANSFECTION AND / OR RAAV VECTOR PRODUCTION
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3068622A1 (en) 2017-06-30 2019-01-03 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
WO2019028192A1 (en) 2017-08-01 2019-02-07 Spark Therapeutics, Inc. METHODS OF GENE THERAPY TARGETING FACTOR VIII (FVIII)
TWI722310B (zh) 2017-08-03 2021-03-21 美商航海家醫療公司 用於aav之遞送之組合物及方法
CN111132993A (zh) 2017-08-29 2020-05-08 海德堡鲁普雷希特卡尔斯大学 合理设计的用于调节嵌合抗原受体(car)-t细胞疗法的病毒样颗粒
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
DK3250239T3 (en) 2019-03-11
US12138318B2 (en) 2024-11-12
WO2016181123A1 (en) 2016-11-17
JP2020096587A (ja) 2020-06-25
EP3250239A1 (en) 2017-12-06
US10414803B2 (en) 2019-09-17
US20200079821A1 (en) 2020-03-12
KR20180019543A (ko) 2018-02-26
AU2016261454A1 (en) 2017-11-23
CN107864653B (zh) 2022-06-21
CA2985945A1 (en) 2016-11-17
US20180111965A1 (en) 2018-04-26
CA2985945C (en) 2022-09-06
LT3250239T (lt) 2019-03-25
PT3250239T (pt) 2019-02-19
PL3250239T3 (pl) 2019-05-31
TW201704253A (zh) 2017-02-01
US20250127922A1 (en) 2025-04-24
EP3250239B1 (en) 2018-12-12
GB201508026D0 (en) 2015-06-24
JP2018520997A (ja) 2018-08-02
HRP20190442T1 (hr) 2019-05-03
CN107864653A (zh) 2018-03-30
JP6619454B2 (ja) 2019-12-11
ES2711349T3 (es) 2019-05-03
TR201901582T4 (tr) 2019-02-21
EP3511021A1 (en) 2019-07-17
AU2016261454B2 (en) 2019-07-11

Similar Documents

Publication Publication Date Title
HUE042693T2 (hu) Kapszid
DK3283625T3 (da) Nukleasemedieret genomeditering
DK3331869T3 (da) Muscarinagonister
DK3277719T3 (da) Polypeptider
EP3316375A4 (en) Redox flow cell
DK3331528T3 (da) Muskarinagonister
DK3305788T3 (da) Janus-kinase-hæmmer
DK3360890T3 (da) Genterapi
DK3331529T3 (da) Muskarinagonister
DK3310450T3 (da) Olie-vand-separator
EP3290364A4 (en) Conveyor belt wear monitoring system
DK3270930T3 (da) Præeklampsi
EP3381654A4 (en) Liquid blow molding method
DK3394281T3 (da) Gærcelle
EP3363322A4 (en) Hair extender
DK3292136T3 (da) Penicillin-g-acylaser
DK3274482T3 (da) Stenborsknop
DE112015006486A5 (de) Inkubationsrinne
EP3377179A4 (en) DEUTERED EPI-743
EP3548009A4 (en) POLYMERIC NANOPARTICLES
DK3318308T3 (da) Badmintonketsjer
DE112015005905A5 (de) Gurtschlossbringer
DE112016005599A5 (de) Strahlungsgrill
ES1140083Y (es) Descompatactador tetrafunción
DE102015206660A8 (de) Plattenaufteilanlage