JP2018520997A5 - - Google Patents

Download PDF

Info

Publication number
JP2018520997A5
JP2018520997A5 JP2017558723A JP2017558723A JP2018520997A5 JP 2018520997 A5 JP2018520997 A5 JP 2018520997A5 JP 2017558723 A JP2017558723 A JP 2017558723A JP 2017558723 A JP2017558723 A JP 2017558723A JP 2018520997 A5 JP2018520997 A5 JP 2018520997A5
Authority
JP
Japan
Prior art keywords
capsid protein
sequence
amino acid
protein according
acid sequence
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
JP2017558723A
Other languages
English (en)
Japanese (ja)
Other versions
JP6619454B2 (ja
JP2018520997A (ja
Filing date
Publication date
Priority claimed from GBGB1508026.0A external-priority patent/GB201508026D0/en
Application filed filed Critical
Publication of JP2018520997A publication Critical patent/JP2018520997A/ja
Publication of JP2018520997A5 publication Critical patent/JP2018520997A5/ja
Application granted granted Critical
Publication of JP6619454B2 publication Critical patent/JP6619454B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2017558723A 2015-05-11 2016-05-10 キャプシド Active JP6619454B2 (ja)

Applications Claiming Priority (3)

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

Related Child Applications (1)

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

Publications (3)

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

Family

ID=53489438

Family Applications (2)

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

Family Applications After (1)

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

Country Status (18)

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

Families Citing this family (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
GB201508026D0 (en) * 2015-05-11 2015-06-24 Ucl Business Plc Capsid
IL314171A (en) 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
GB201608046D0 (en) 2016-05-09 2016-06-22 Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The Treatment of complement-mediated disorders
KR102863181B1 (ko) 2016-12-30 2025-09-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 윌슨병을 치료하기 위한 유전자 치료
CN111511375A (zh) * 2017-06-30 2020-08-07 因提玛生物科学公司 用于基因治疗的腺相关病毒载体
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
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
CA3095179A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
KR102114249B1 (ko) 2018-08-21 2020-05-22 코나아이 (주) 가상 화폐 결제 방법 및 시스템
GB201817810D0 (en) 2018-10-31 2018-12-19 Ucl Business Plc Modified factor IX polypeptides
KR20210112339A (ko) 2019-01-04 2021-09-14 울트라제닉스 파마수티컬 인코포레이티드 윌슨병을 치료하기 위한 유전자 요법 구축물
CN113727992A (zh) * 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
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
CA3136545A1 (en) 2019-04-12 2020-10-15 Freeline Therapeutics Limited Plasmid system
PL3722434T3 (pl) 2019-04-12 2022-12-12 Freeline Therapeutics Limited Układ plazmidowy
JP7371954B2 (ja) * 2019-07-12 2023-10-31 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
JP7785352B2 (ja) * 2019-08-14 2025-12-15 ユニバーシティー オブ フロリダ リサーチ ファンデーション, インク. 遺伝子治療のためのaavカプシドバリアント
AU2020336332A1 (en) * 2019-08-29 2022-03-03 The Board Of Trustees Of The Leland Stanford Junior University Adeno-associated viral vectors for crossing the human blood brain barrier
MX2022004353A (es) 2019-10-17 2022-07-19 Casete de transferencia de aav.
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
TW202142554A (zh) * 2020-02-25 2021-11-16 澳洲兒童醫學研究所 腺相關病毒蛋白殼多肽及載體
US20230242905A1 (en) * 2020-07-03 2023-08-03 Genethon Method for Engineering Novel Hybrid AAV Capsids Through Hyper Variable Regions Swapping
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
JP2023542614A (ja) 2020-08-19 2023-10-11 サレプタ セラピューティクス, インコーポレイテッド レット症候群の治療のためのアデノ随伴ウイルスベクター
EP4214242A1 (en) 2020-09-15 2023-07-26 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
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
AU2021371307A1 (en) 2020-10-28 2023-06-01 Regenxbio, Inc. VECTORIZED ANTI-TNF-α ANTIBODIES FOR OCULAR INDICATIONS
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
WO2023023781A1 (en) * 2021-08-25 2023-03-02 Children's Medical Research Institute Modified aav capsid polypeptides and vectors
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
TW202400803A (zh) 2022-05-03 2024-01-01 美商銳進科斯生物股份有限公司 載體化抗補體抗體與補體劑及其投與
TW202417633A (zh) 2022-05-03 2024-05-01 美商銳進科斯生物股份有限公司 用於眼適應症之載體化抗TNF-α抑制劑
GB202214328D0 (en) 2022-09-29 2022-11-16 Freeline Therapeutics Ltd Method
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
EP4752226A1 (en) 2024-11-28 2026-06-03 Ascend Advanced Therapies GmbH Plasmid system for aav production
WO2026109693A1 (en) 2024-11-21 2026-05-28 Ascend Advanced Therapies Ltd Method for improved aav production

Family Cites Families (101)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
AU1449499A (en) 1997-10-31 1999-05-24 Maxygen, Incorporated Modification of virus tropism and host range by viral genome shuffling
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
EP1038959A1 (en) 1999-03-17 2000-09-27 Aventis Behring Gesellschaft mit beschränkter Haftung Factor VIII without B-domain, comprising one or more insertions of a truncated intron I of factor IX
EP1048726B1 (en) 1999-04-27 2006-07-26 Négrier, Claude Modified factor VIII cDNA
EP1228234A2 (en) * 2000-03-14 2002-08-07 Neurologix, Inc. Production of chimeric capsid vectors
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
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
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
US7858367B2 (en) 2002-04-30 2010-12-28 Duke University Viral vectors and methods for producing and using the same
PL2657248T3 (pl) 2003-06-19 2017-09-29 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP1668143B1 (en) 2003-09-30 2013-03-20 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
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
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
WO2008024724A1 (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
DK2623605T3 (en) 2007-04-09 2019-03-25 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
EP2158211B1 (en) * 2007-05-31 2016-08-10 Medigene AG Mutated structural protein of a parvovirus
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
CA2754335C (en) 2009-03-04 2019-12-24 Medigene Ag 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
EP3795581A3 (en) 2011-08-24 2021-06-09 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
EP2771455B1 (en) 2011-10-28 2016-10-05 The University of North Carolina At Chapel Hill Cell line for production of adeno-associated virus
PL2900686T3 (pl) 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
DK2911687T3 (da) 2012-10-26 2019-05-13 Univ Brussel Vrije Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf
WO2014159546A1 (en) 2013-03-14 2014-10-02 Asklepios Biopharmaceutical, Inc. Modified soluble vegf receptor-1 genes and vectors for gene therapy
ES2926774T3 (es) 2013-03-15 2022-10-28 Univ North Carolina Chapel Hill Métodos y composiciones para vectores AAV de doble unión de glicano
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
NZ716102A (en) 2013-07-22 2021-12-24 The Children’S Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
RS63942B1 (sr) 2013-08-27 2023-02-28 Res Inst Nationwide Childrens Hospital Proizvodi i postupci za lečenje amiotrofične lateralne skleroze
HUE052676T2 (hu) * 2013-10-11 2021-05-28 Massachusetts Eye & Ear Infirmary Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik
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
EP3113787B1 (en) 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
ES2744565T3 (es) 2014-04-25 2020-02-25 Genethon Tratamiento de la hiperbilirrubinemia
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
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
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
US20180245073A1 (en) 2015-02-23 2018-08-30 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
FI3265178T3 (fi) 2015-03-02 2026-08-03 Adverum Biotechnologies Inc Koostumuksia ja menetelmiä polynukleotidien viemiseksi intravitreaalisesti retinaalisiin tappisoluihin
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
EP3285788B1 (en) 2015-04-23 2024-12-18 University of Massachusetts Modulation of aav vector transgene expression
GB201508026D0 (en) * 2015-05-11 2015-06-24 Ucl Business Plc Capsid
EP4378487A3 (en) 2015-06-23 2024-09-04 The Children's Hospital of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
WO2017015102A1 (en) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
WO2017023724A1 (en) 2015-07-31 2017-02-09 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
IL314171A (en) 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
WO2017066764A2 (en) 2015-10-16 2017-04-20 William Marsh Rice University Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
AU2016343979A1 (en) 2015-10-29 2018-05-10 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
PE20231949A1 (es) 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
SG10201912763QA (en) 2015-11-05 2020-02-27 Bamboo Therapeutics Inc Modified friedreich ataxia genes and vectors for gene therapy
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
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
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
US11124546B2 (en) 2015-12-14 2021-09-21 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
CA3014637A1 (en) 2016-02-16 2017-08-24 Mark A. Kay Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
AU2017234929B2 (en) 2016-03-18 2024-05-02 The Children's Hospital Of Philadelphia Therapeutic for treatment of diseases including the central nervous system
US11433116B2 (en) 2016-04-14 2022-09-06 Cedars-Sinai Medical Center GJA1 isoforms protect against metabolic stress
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
WO2017189963A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
ES3066293T3 (en) 2016-06-16 2026-05-12 Charite Univ Berlin Koerperschaft Des Oeffentlichen Rechts Neuropeptide-expressing vectors and methods for the treatment of epilepsy
JP6994018B2 (ja) 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド 新規アデノ随伴ウイルスキャプシドタンパク質
HUE053747T2 (hu) 2016-08-15 2021-07-28 Genzyme Corp Eljárások AAV kimutatására
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
KR102617502B1 (ko) 2016-09-12 2023-12-26 제네똥 산-알파 글루코시다제 변이체 및 이의 용도
EP3293203A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
KR20190075964A (ko) 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 Aav 캡시드 설계
EP3532075A4 (en) 2016-10-27 2020-07-08 Intima Bioscience, Inc. VIRAL METHODS OF MANUFACTURING GENETICALLY MODIFIED CELLS
EP3583220A4 (en) 2017-02-15 2021-07-07 The University of North Carolina at Chapel Hill PROCEDURES AND COMPOSITIONS FOR GENERAL TRANSFER IN THE VASCULAR SYSTEM
MA47820A (fr) 2017-03-10 2020-01-15 Genethon Traitement de la glycogénose de type iii
WO2018170310A1 (en) 2017-03-15 2018-09-20 The University Of North Carolina At Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
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
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
WO2018226887A1 (en) 2017-06-07 2018-12-13 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
EP3658250A4 (en) 2017-06-30 2021-10-27 Spark Therapeutics, Inc. AAV VECTOR COLUMN CLEANING PROCEDURE
CA3071769A1 (en) 2017-07-31 2019-02-07 Reflection Biotechnologies Limited Cellular models of and therapies for ocular diseases
US20200237930A1 (en) 2017-08-01 2020-07-30 Spark Therapeutics, Inc. Factor viii (fviii) gene therapy methods
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
JP7329254B2 (ja) 2017-08-29 2023-08-18 ルプレヒト-カールス-ウニヴェルジテート ハイデルベルク キメラ抗原受容体(car)-t細胞療法の調節のための合理的に設計されたウイルス様粒子
US20200377887A1 (en) 2017-09-22 2020-12-03 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
US12123027B2 (en) 2017-10-12 2024-10-22 Ascend Advanced Therapies Limited Life-cycle-defective adenovirus helper viruses, their production and use for producing rAAV

Similar Documents

Publication Publication Date Title
JP2019530462A5 (enExample)
JP2016538885A5 (enExample)
JP2017048194A5 (enExample)
JP2020022459A5 (enExample)
CL2019002280A1 (es) Vectores virales, adenovirales y composiciones a base de una secuencia de ornitina transcarbamilasa humana (hotch) modificada genéticamente y su uso en el tratamiento de deficiencia de la otc. (divisional solicitud 201602235)
JP2016537341A5 (enExample)
JP2014012022A5 (enExample)
JP2018515096A5 (enExample)
EP4378487A3 (en) Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
JP2019513794A5 (enExample)
JP2018537087A5 (enExample)
JP2020514286A5 (enExample)
JP2016512683A5 (enExample)
JP2018528253A5 (enExample)
JP2017532966A5 (enExample)
JP2014503206A5 (enExample)
JP2015096070A5 (enExample)
JP2014516536A5 (enExample)
JP2013502225A5 (enExample)
FI3872085T3 (fi) Esimuotoiset virussekvenssit ja niiden käytöt
RU2016123086A (ru) Способы и композиции для лечения болезни хантингтона
JP2016513115A5 (enExample)
JP2017536116A5 (enExample)
JP2019523648A5 (enExample)
JP2017500061A5 (enExample)