JP2014012022A5 - - Google Patents

Download PDF

Info

Publication number
JP2014012022A5
JP2014012022A5 JP2013191907A JP2013191907A JP2014012022A5 JP 2014012022 A5 JP2014012022 A5 JP 2014012022A5 JP 2013191907 A JP2013191907 A JP 2013191907A JP 2013191907 A JP2013191907 A JP 2013191907A JP 2014012022 A5 JP2014012022 A5 JP 2014012022A5
Authority
JP
Japan
Prior art keywords
aav
seq
adeno
capsid protein
associated virus
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
JP2013191907A
Other languages
English (en)
Japanese (ja)
Other versions
JP2014012022A (ja
JP5911069B2 (ja
Filing date
Publication date
Application filed filed Critical
Publication of JP2014012022A publication Critical patent/JP2014012022A/ja
Publication of JP2014012022A5 publication Critical patent/JP2014012022A5/ja
Application granted granted Critical
Publication of JP5911069B2 publication Critical patent/JP5911069B2/ja
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

Links

JP2013191907A 2003-09-30 2013-09-17 アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途 Expired - Lifetime JP5911069B2 (ja)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US50822603P 2003-09-30 2003-09-30
US60/508,226 2003-09-30
US56654604P 2004-04-29 2004-04-29
US60/566,546 2004-04-29

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2010272649A Division JP5797397B2 (ja) 2003-09-30 2010-12-07 アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2015114170A Division JP6335141B2 (ja) 2003-09-30 2015-06-04 アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途

Publications (3)

Publication Number Publication Date
JP2014012022A JP2014012022A (ja) 2014-01-23
JP2014012022A5 true JP2014012022A5 (enExample) 2014-10-02
JP5911069B2 JP5911069B2 (ja) 2016-04-27

Family

ID=34426044

Family Applications (9)

Application Number Title Priority Date Filing Date
JP2006533879A Expired - Lifetime JP5054975B2 (ja) 2003-09-30 2004-09-30 アデノ随伴ウイルス(aav)の同源系統群、配列、それらを含有するベクターおよびそれらの用途
JP2010272649A Expired - Lifetime JP5797397B2 (ja) 2003-09-30 2010-12-07 アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途
JP2012053003A Expired - Lifetime JP5431516B2 (ja) 2003-09-30 2012-03-09 アデノ随伴ウイルス(aav)の同源系統群、配列、それらを含有するベクターおよびそれらの用途
JP2013191907A Expired - Lifetime JP5911069B2 (ja) 2003-09-30 2013-09-17 アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途
JP2015114170A Expired - Lifetime JP6335141B2 (ja) 2003-09-30 2015-06-04 アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途
JP2016211386A Expired - Lifetime JP6516713B2 (ja) 2003-09-30 2016-10-28 アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途
JP2018067535A Expired - Lifetime JP6673963B2 (ja) 2003-09-30 2018-03-30 アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途
JP2019221986A Pending JP2020054373A (ja) 2003-09-30 2019-12-09 アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途
JP2022161483A Pending JP2022173573A (ja) 2003-09-30 2022-10-06 アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途

Family Applications Before (3)

Application Number Title Priority Date Filing Date
JP2006533879A Expired - Lifetime JP5054975B2 (ja) 2003-09-30 2004-09-30 アデノ随伴ウイルス(aav)の同源系統群、配列、それらを含有するベクターおよびそれらの用途
JP2010272649A Expired - Lifetime JP5797397B2 (ja) 2003-09-30 2010-12-07 アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途
JP2012053003A Expired - Lifetime JP5431516B2 (ja) 2003-09-30 2012-03-09 アデノ随伴ウイルス(aav)の同源系統群、配列、それらを含有するベクターおよびそれらの用途

Family Applications After (5)

Application Number Title Priority Date Filing Date
JP2015114170A Expired - Lifetime JP6335141B2 (ja) 2003-09-30 2015-06-04 アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途
JP2016211386A Expired - Lifetime JP6516713B2 (ja) 2003-09-30 2016-10-28 アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途
JP2018067535A Expired - Lifetime JP6673963B2 (ja) 2003-09-30 2018-03-30 アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途
JP2019221986A Pending JP2020054373A (ja) 2003-09-30 2019-12-09 アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途
JP2022161483A Pending JP2022173573A (ja) 2003-09-30 2022-10-06 アデノ随伴ウイルス(aav)の同源系統群(クレイド)、配列、それらを含有するベクターおよびそれらの用途

Country Status (17)

Country Link
US (13) US7906111B2 (enExample)
EP (7) EP2298926A1 (enExample)
JP (9) JP5054975B2 (enExample)
CN (4) CN1856576B (enExample)
AU (2) AU2004278684B2 (enExample)
BE (1) BE2021C533I2 (enExample)
CA (3) CA2537793C (enExample)
CY (3) CY1119833T1 (enExample)
DK (4) DK2292780T3 (enExample)
ES (5) ES2555612T3 (enExample)
FR (1) FR21C1037I1 (enExample)
HU (4) HUE035567T2 (enExample)
NZ (1) NZ545628A (enExample)
PL (2) PL2292780T3 (enExample)
PT (2) PT3211085T (enExample)
SI (2) SI3211085T1 (enExample)
WO (1) WO2005033321A2 (enExample)

Families Citing this family (635)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2915124C (en) 2001-11-13 2018-08-14 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2602352T3 (es) 2001-12-17 2017-02-20 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas
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
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
WO2006078279A2 (en) 2004-04-28 2006-07-27 The Trustees Of The University Of Pennsylvania Sequential delivery of immunogenic molecules via adenovirus and adeno-associated virus-mediated administrations
US9592284B2 (en) 2004-04-28 2017-03-14 The Trustees Of The University Of Pennsylvania Immunization regimen with E4-deleted adenovirus prime and E1-deleted adenovirus boost
EP2357010B1 (en) 2005-04-07 2013-06-12 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
EP2009990B1 (en) 2006-04-07 2016-09-21 The Board of Regents of The University of Texas System Methods and compositions related to adenoassociated virus-phage particles
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
US20090317417A1 (en) * 2006-04-28 2009-12-24 The Trustees Of The University Of Pennsylvania Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof
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
HUE030719T2 (en) 2007-04-09 2017-05-29 Univ Florida RAAV vector formulations and methods comprising tyrosine-modified capsid proteins for their use
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
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
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
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
HUE044522T2 (hu) 2009-04-30 2019-10-28 Univ Pennsylvania Adeno-asszociált vírus konstrukciókat tartalmazó légúti vezetõ sejtek célzására szolgáló kompozíciók
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2010141706A1 (en) * 2009-06-03 2010-12-09 Cedars-Sinai Medical Center Effective vector platform for gene transfer and gene therapy
US20120244127A1 (en) 2009-10-01 2012-09-27 The Trustees Of The University Of Pennsylvania AAV Vectors Expressing SEC10 for Treating Kidney Damage
SG10201502270TA (en) 2010-03-29 2015-05-28 Univ Pennsylvania Pharmacologically induced transgene ablation system
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
EP3514232A1 (en) 2010-04-23 2019-07-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
JP2013533847A (ja) 2010-04-23 2013-08-29 ユニバーシティ オブ マサチューセッツ コレステロール関連障害のaavベースの治療
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
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
CN107828820B (zh) * 2010-10-27 2022-06-07 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
EP4234571A3 (en) * 2011-02-10 2023-09-27 The University of North Carolina at Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
JP6224459B2 (ja) 2011-02-17 2017-11-01 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 組織特異性を改変し、aav9媒介遺伝子導入を改善するための組成物および方法
WO2012145572A1 (en) 2011-04-20 2012-10-26 The Trustees Of The University Of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
WO2012145624A2 (en) 2011-04-21 2012-10-26 University Of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
RS66184B1 (sr) 2011-04-22 2024-12-31 Univ California Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu
US20130039888A1 (en) * 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
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
WO2013052915A2 (en) 2011-10-05 2013-04-11 Genelux Corporation Method for detecting replication or colonization of a biological therapeutic
US9226936B2 (en) 2011-10-28 2016-01-05 The Wistar of Anatomy and Biology Methods and compositions for enhancing the therapeutic effect of anti-tumor T cells
WO2013082268A1 (en) 2011-11-30 2013-06-06 The Wistar Institute Of Anatomy And Biology Methods and compositions for regulation of cell aging, carcinogenesis, and reprogramming
EP2601968A1 (en) * 2011-12-06 2013-06-12 Deutsches Krebsforschungszentrum HPV derived polynucleic acids for therapy
US9611305B2 (en) 2012-01-06 2017-04-04 Mayo Foundation For Medical Education And Research Treating cardiovascular or renal diseases
US20140087362A1 (en) 2012-03-16 2014-03-27 Aladar A. Szalay Methods for assessing effectiveness and monitoring oncolytic virus treatment
US20130280170A1 (en) 2012-04-20 2013-10-24 Aladar A. Szalay Imaging methods for oncolytic virus therapy
EP2847337A4 (en) * 2012-05-09 2016-04-27 Univ Oregon Health & Science ADENO ASSOCIATED VIRUS PLASMIDS AND VECTORS
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
CA2878171C (en) 2012-07-11 2021-04-27 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
EP3769789A1 (en) 2012-08-01 2021-01-27 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
JP6387350B2 (ja) 2012-09-28 2018-09-05 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 希突起神経膠細胞を標的とするアデノ随伴ウイルスベクター
WO2014055960A1 (en) 2012-10-05 2014-04-10 Genelux Corporation Energy absorbing-based diagnostic and therapeutic methods employing nucleic acid molecules encoding chromophore-producing enzymes
EP2749569A1 (en) 2012-12-28 2014-07-02 Annibale Alessandro Puca Variant of BPIFB4 protein
US8957044B2 (en) 2013-03-01 2015-02-17 Wake Forest University Health Sciences Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
EP2970946A4 (en) * 2013-03-13 2016-09-07 Philadelphia Children Hospital ADENOASSOZED VIRUS VECTORS AND METHOD FOR USE THEREOF
EP2970884A4 (en) 2013-03-14 2016-11-02 Univ Florida DI-AMINO ACID-REINFORCED PROTEINS ASSOCIATED WITH ALS
CA2904396A1 (en) * 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav vectors
EP3747998B1 (en) 2013-03-15 2024-07-24 The Trustees of the University of Pennsylvania Compositions for treating mpsi
WO2015012924A2 (en) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof
ES2897508T3 (es) 2013-05-31 2022-03-01 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
JP6985795B2 (ja) 2013-09-26 2021-12-22 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー
EP3054995A1 (en) * 2013-10-07 2016-08-17 Kiromic, LLC Compositions and methods for treating cardiovascular diseases using disease-specific promoter
JP6552511B2 (ja) * 2013-10-11 2019-07-31 マサチューセッツ アイ アンド イヤー インファーマリー 祖先ウイルス配列を予測する方法およびその使用
US10238700B2 (en) 2014-01-02 2019-03-26 Genelux Corporation Oncolytic virus adjunct therapy with agents that increase virus infectivity
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
RS60902B1 (sr) 2014-03-09 2020-11-30 Univ Pennsylvania Kompozicije korisne u lečenju nedostatka ornitinske transkarbamilaze (otc)
PL3117005T3 (pl) 2014-03-10 2024-11-04 Uniqure Ip B.V. Dodatkowo ulepszone wektory aav produkowane w komórkach owadzich
KR102288849B1 (ko) 2014-03-17 2021-08-12 애드베룸 바이오테크놀로지스, 인코포레이티드 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법
DK3119797T3 (da) 2014-03-18 2021-03-15 Univ Massachusetts Raav-baserede sammensætninger og fremgangsmåder til behandling af amyotrofisk lateralsklerose
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
WO2015164778A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennysylvania Ldlr variants and their use in compositions for reducing cholesterol levels
US10780182B2 (en) 2014-04-25 2020-09-22 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
US10975391B2 (en) 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
SG11201609207SA (en) 2014-05-13 2016-12-29 Univ Pennsylvania Compositions comprising aav expressing dual antibody constructs and uses thereof
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
EP3800260A1 (en) 2014-09-24 2021-04-07 City of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
US10711270B2 (en) 2014-10-03 2020-07-14 University Of Massachusetts High efficiency library-identified AAV vectors
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
EP3207130B1 (en) 2014-10-14 2019-08-07 Halozyme, Inc. Compositions of adenosine deaminase-2 (ada2), variants thereof and methods of using same
RU2020140209A (ru) 2014-10-21 2021-01-25 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
WO2016073739A1 (en) * 2014-11-05 2016-05-12 Research Institute At Nationwide Children's Hospital Methods and materials for producing recombinant viruses in eukaryotic microalgae
CN107106689A (zh) * 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
KR102584655B1 (ko) 2014-11-14 2023-10-06 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
RU2727015C2 (ru) * 2014-11-21 2020-07-17 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Векторы aav, нацеленные на центральную нервную систему
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
JP6754361B2 (ja) 2014-12-16 2020-09-09 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法
ES2900973T3 (es) 2015-01-07 2022-03-21 UNIV AUTòNOMA DE BARCELONA Constructo genético de vector individual que comprende genes de insulina y glucoquinasa
AU2016206518B2 (en) 2015-01-16 2020-03-05 University Of Washington Novel micro-dystrophins and related methods of use
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
EP3256487A4 (en) 2015-02-09 2018-07-18 Duke University Compositions and methods for epigenome editing
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
KR20170137730A (ko) 2015-03-02 2017-12-13 애드베룸 바이오테크놀로지스, 인코포레이티드 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법
AU2016229000B2 (en) 2015-03-10 2021-11-04 The Trustees Of Columbia University In The City Of New York Recombinant Glut1 adeno-associated viral vector constructs and related methods for restoring Glut1 expression
EP3277819B1 (en) * 2015-03-24 2021-03-03 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
WO2016172008A1 (en) 2015-04-24 2016-10-27 University Of Massachusetts Modified aav constructions and uses thereof
WO2016176191A1 (en) 2015-04-27 2016-11-03 The Trustees Of The University Of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
EP3291765A4 (en) 2015-05-07 2019-01-23 Massachusetts Eye & Ear Infirmary METHOD FOR THE DELIVERY OF AN ACTIVE SUBSTANCE TO THE EYE
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
CA2985786A1 (en) * 2015-05-12 2016-11-17 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
CN108135994B (zh) 2015-05-13 2022-01-25 宾夕法尼亚州大学信托人 Aav-介导的抗-流感抗体的表达及其使用方法
US20170067028A1 (en) * 2015-05-15 2017-03-09 Douglas J. Ballon Radiolabeling of adeno associated virus
WO2016196324A1 (en) 2015-05-29 2016-12-08 University Of Floridia Research Foundation, Inc. Methods for diagnosing huntington's disease
WO2017015637A1 (en) 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
LT3872085T (lt) * 2015-07-30 2023-05-10 Massachusetts Eye & Ear Infirmary Viruso prototipo sekos ir jų panaudojimas
GB2557123B (en) 2015-07-31 2021-11-03 Univ Minnesota Modified cells and methods of therapy
EP3332012A4 (en) 2015-08-06 2019-01-16 The Trustees of The University of Pennsylvania GLP-1 AND USE THEREOF IN COMPOSITIONS FOR THE TREATMENT OF METABOLISM DISEASES
JP6905755B2 (ja) 2015-08-25 2021-07-21 デューク ユニバーシティ Rnaガイド型エンドヌクレアーゼを使用してゲノム工学における特異性を改善する組成物および方法
US11117942B2 (en) 2015-08-31 2021-09-14 The Trustees Of The University Of Pennsylvania AAV-EPO for treating companion animals
RU2727411C2 (ru) 2015-09-24 2020-07-21 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Композиция и способ для лечения заболевания, опосредованного комплементом
EP3831842A1 (en) 2015-09-28 2021-06-09 The University of North Carolina at Chapel Hill Methods and compositions for antibody-evading virus vectors
WO2017062750A1 (en) 2015-10-09 2017-04-13 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
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
US11426469B2 (en) 2015-10-22 2022-08-30 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
CA3002982A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
WO2017070491A1 (en) * 2015-10-23 2017-04-27 Applied Genetic Technologies Corporation Ophthalmic formulations
WO2017075119A1 (en) 2015-10-28 2017-05-04 The Trustees Of The Univeresity Of Pennsylvania Intrathecal administration of adeno-associated-viral vectors for gene therapy
EP3368054A4 (en) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. REGULATORY EXPRESSION USING THE ADENO-ASSOCIATED VIRUS (AAV)
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
US10983110B2 (en) 2015-12-02 2021-04-20 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
FR3044926B1 (fr) 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
WO2017160360A2 (en) 2015-12-11 2017-09-21 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
EP3400304B1 (en) 2015-12-11 2022-04-06 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
WO2017100704A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
ES2934848T3 (es) 2015-12-11 2023-02-27 Univ Pennsylvania Método de purificación escalable para AAV8
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
JP6990182B2 (ja) 2015-12-11 2022-02-15 マサチューセッツ アイ アンド イヤー インファーマリー 蝸牛および前庭細胞に核酸を送達するための材料および方法
JP7082050B2 (ja) * 2015-12-14 2022-06-07 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 脊髄性筋萎縮症の処置において有用なアデノ-関連ウイルスベクター
EP3389724B1 (en) 2015-12-14 2020-07-01 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
US11241506B2 (en) 2015-12-14 2022-02-08 The Trustees Of The University Of Pennsylvania Composition for treatment of Crigler-Najjar syndrome
US11826433B2 (en) 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
US10973929B2 (en) 2016-02-03 2021-04-13 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type I
IL300254B2 (en) 2016-02-05 2025-06-01 Univ Emory Administration of single stranded or self-complementary adeno-associated virus 9 by injection into the cerebrospinal fluid to target gene therapy to the central nervous system
CA3012344A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
US10940161B2 (en) 2016-04-04 2021-03-09 University Of Florida Research Foundation, Incorporated Manipulation of EIF3 to modulate repeat associated non-ATG (RAN) translation
WO2017176929A1 (en) 2016-04-05 2017-10-12 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
WO2017180587A2 (en) 2016-04-11 2017-10-19 Obsidian Therapeutics, Inc. Regulated biocircuit systems
WO2017180915A2 (en) 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
AU2017248659B2 (en) 2016-04-15 2022-08-11 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia A
WO2017181113A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
EP3443000B1 (en) 2016-04-15 2025-11-12 Alpine Immune Sciences, Inc. Cd80 variant immunomodulatory proteins and uses thereof
KR102824067B1 (ko) 2016-04-15 2025-06-23 알파인 이뮨 사이언시즈, 인코포레이티드 Icos 리간드 변이체 면역조절 단백질 및 그의 용도
CN109661470A (zh) 2016-04-15 2019-04-19 宾夕法尼亚州大学信托人 新型aav8突变衣壳和含有其的组合物
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
MA44873A (fr) 2016-04-15 2019-03-13 Univ Pennsylvania Composition pour le traitement de la dégénérescence maculaire liée a l'âge exsudative
WO2017184463A1 (en) 2016-04-17 2017-10-26 The Trustees Of The University Of Pennsylvania Compositions and methods useful for prophylaxis of organophosphates
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
AU2017261249B2 (en) * 2016-05-03 2021-05-06 Children's Medical Research Institute Adeno-associated virus polynucleotides, polypeptides and virions
CN109476707B (zh) 2016-05-13 2022-12-02 4D分子治疗有限公司 腺相关病毒变体衣壳和其使用方法
AU2017268382B2 (en) 2016-05-18 2023-09-28 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
RU2758488C2 (ru) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
JP2019517274A (ja) 2016-06-13 2019-06-24 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The University Of North Carolina At Chapel Hill 最適化されたcln1遺伝子および発現カセットおよびそれらの使用
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
EP3481958A4 (en) 2016-07-08 2019-12-25 The Trustees of The University of Pennsylvania METHOD AND COMPOSITIONS FOR TREATING DISTURBANCES AND DISEASES WITH RDH12
EP3487523B1 (en) 2016-07-19 2023-09-06 Duke University Therapeutic applications of cpf1-based genome editing
US11883470B2 (en) 2016-07-25 2024-01-30 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
IL263801B2 (en) 2016-07-26 2024-01-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
CN110088127A (zh) 2016-07-28 2019-08-02 高山免疫科学股份有限公司 Cd155变体免疫调节蛋白及其用途
US11834490B2 (en) 2016-07-28 2023-12-05 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
JP7071332B2 (ja) 2016-07-29 2022-05-18 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 変異カプシドを有するアデノ随伴ウイルスビリオン及びその使用方法
IL264872B2 (en) 2016-08-18 2025-02-01 Univ California CRISPR-CAS genome engineering using a modular AAV delivery system
JP2019531787A (ja) 2016-08-30 2019-11-07 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 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
US10457940B2 (en) * 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
CN110520530A (zh) 2016-10-18 2019-11-29 明尼苏达大学董事会 肿瘤浸润性淋巴细胞和治疗方法
US11192925B2 (en) 2016-10-19 2021-12-07 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
AU2017345764A1 (en) 2016-10-20 2019-05-02 Alpine Immune Sciences, Inc. Secretable variant immunomodulatory proteins and engineered cell therapy
WO2018083606A1 (en) 2016-11-01 2018-05-11 Novartis Ag Methods and compositions for enhancing gene editing
EP3548065B1 (en) 2016-12-01 2022-11-09 INSERM - Institut National de la Santé et de la Recherche Médicale Pharmaceutical compositions for the treatment of retinal degenerative diseases
WO2018126112A1 (en) 2016-12-30 2018-07-05 The Trustees Of The University Of Pennsylvania Gene therapy for treating phenylketonuria
MX2019007873A (es) 2016-12-30 2019-11-18 Univ Pennsylvania Terapia génica para tratar la enfermedad de wilson.
WO2018134168A1 (en) 2017-01-17 2018-07-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors
EP3576760A2 (en) 2017-02-01 2019-12-11 The Trustees Of The University Of Pennsylvania Gene therapy for treating citrullenemia
AU2018219895C1 (en) 2017-02-09 2024-10-03 Indapta Therapeutics, Inc. Engineered Natural Killer (NK) cells and compositions and methods thereof
US11554147B2 (en) 2017-02-20 2023-01-17 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
CA3114549A1 (en) 2017-02-21 2018-08-30 University Of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof
WO2018154096A1 (en) 2017-02-24 2018-08-30 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Method for re-expression of different hypermethylated genes involved in fibrosis, like hypermethylated rasal,1 and use thereof in treatment of fibrosis as well as kit of parts for re-expression of hypermethylated genes including rasal1 in a subject
US11827906B2 (en) * 2017-02-28 2023-11-28 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clade f vector and uses therefor
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
SG11201907611WA (en) 2017-02-28 2019-09-27 Univ Pennsylvania Influenza vaccines based on aav vectors
CN119752819A (zh) 2017-03-01 2025-04-04 宾夕法尼亚州立大学托管会 用于眼部病症的基因疗法
EP3589311A1 (en) 2017-03-02 2020-01-08 Genethon Method for removing anti-aav antibodies from a blood-derived composition
CA3054068A1 (en) 2017-03-16 2018-09-20 Alpine Immune Sciences, Inc. Cd80 variant immunomodulatory proteins and uses thereof
LT3596116T (lt) 2017-03-16 2023-11-10 Alpine Immune Sciences, Inc. Imunomoduliacinių baltymų pd-l1 variantai ir jų naudojimo būdai
CN110809581A (zh) 2017-03-16 2020-02-18 高山免疫科学股份有限公司 Pd-l2变体免疫调节蛋白及其用途
CA3056249A1 (en) 2017-03-24 2018-09-27 Lankenau Institute For Medical Research Methods and compositions for inducible extracellular membrane capture of monoclonal immunoglobulins secreted by hybridomas
CN110770344B (zh) 2017-03-31 2024-09-17 北京三诺佳邑生物技术有限责任公司 shRNA表达框、携带其的多核苷酸序列及其应用
EP3610016A1 (en) 2017-04-10 2020-02-19 Genethon Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies
WO2018191666A1 (en) 2017-04-14 2018-10-18 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2 sulfatase (ids) produced by human neural or glial cells
JP7526455B2 (ja) * 2017-04-17 2024-08-01 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. PKRおよびeIF2A-P経路によるRANタンパク質翻訳の調節
WO2018200542A1 (en) 2017-04-24 2018-11-01 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
AU2018261790B2 (en) 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
WO2018204803A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
JP7327803B2 (ja) 2017-05-09 2023-08-16 ユニバーシティ オブ マサチューセッツ 筋萎縮性側索硬化症(als)を処置する方法
JP7330899B2 (ja) 2017-05-10 2023-08-22 マサチューセッツ アイ アンド イヤー インファーマリー ウイルスのアセンブリ活性化タンパク質(aap)依存性を改変する方法および組成物
RU2019139555A (ru) 2017-05-11 2021-06-11 Зе Трастис Оф Зе Юниверсити Оф Пеннсильвания Генная терапия нейрональных цероидных липофусцинозов
WO2018218359A1 (en) 2017-05-31 2018-12-06 The Trustees Of The University Of Pennsylvania Gene therapy for treating peroxisomal disorders
EP3638316A4 (en) 2017-06-14 2021-03-24 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR EYE DISEASES
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2019006418A2 (en) * 2017-06-30 2019-01-03 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
US20200121746A1 (en) 2017-06-30 2020-04-23 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
JP2020526514A (ja) 2017-07-06 2020-08-31 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 ムコ多糖症i型を治療するための遺伝子治療
FI3648783T3 (fi) 2017-07-07 2024-09-10 Genethon Uudet polynukleotidit, jotka koodaavat ihmisen FKRP-proteiinia
CN111132626B (zh) 2017-07-17 2024-01-30 沃雅戈治疗公司 轨迹阵列引导系统
BR112020001060A2 (pt) 2017-07-27 2020-07-14 Novartis Ag variantes de trem2 resistentes a shedase
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US11680249B2 (en) 2017-08-28 2023-06-20 The Regents Of The University Of California Adeno-associated virus capsid variants and methods of use thereof
FI3684423T3 (fi) 2017-09-20 2023-06-15 4D Molecular Therapeutics Inc Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä
CN111448321A (zh) 2017-09-22 2020-07-24 马萨诸塞大学 Sod1双表达载体及其用途
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
JP7350337B2 (ja) 2017-09-26 2023-09-26 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. 神経障害の処置においてranタンパク質レベルを低下させるためのメトホルミンおよびそのアナログの使用
EP3687582A4 (en) 2017-09-29 2021-07-14 Voyager Therapeutics, Inc. RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
MX2020004540A (es) 2017-10-18 2020-08-03 Alpine Immune Sciences Inc Proteinas inmunomoduladoras de ligando icos variante y composiciones y metodos relacionados.
AU2018350992A1 (en) 2017-10-18 2020-05-21 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
AU2018350990A1 (en) 2017-10-18 2020-05-21 Regenxbio Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified VEGF-Trap
WO2019077159A1 (en) 2017-10-20 2019-04-25 INSERM (Institut National de la Santé et de la Recherche Médicale) METHODS OF EXPRESSING POLYNUCLEOTIDE OF INTEREST IN CONE PHOTORECCEPTORS OF A SUBJECT COMPRISING SUB-RETINAL ADMINISTRATION OF A THERAPEUTICALLY EFFECTIVE AMOUNT OF A RECOMBINANT AAV9 DERIVATIVE VECTOR
AU2018372235B9 (en) 2017-11-27 2022-03-10 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis
JP7389744B2 (ja) 2017-11-30 2023-11-30 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 ムコ多糖症iiia型のための遺伝子療法
CA3083761A1 (en) 2017-11-30 2019-06-06 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis iiib
JP2021506861A (ja) 2017-12-19 2021-02-22 アコーオス インコーポレイテッド 内耳への治療用抗体のaav媒介送達
EP3735417A1 (en) 2018-01-03 2020-11-11 Alpine Immune Sciences, Inc. Multi-domain immunomodulatory proteins and methods of use thereof
WO2019143803A1 (en) 2018-01-17 2019-07-25 Adrenas Therapeutics, Inc. Adeno-associated virus gene therapy for 21-hydroxylase deficiency
BR112020015798A2 (pt) 2018-02-01 2021-03-09 Homology Medicines, Inc. Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer 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
EP3758724A4 (en) * 2018-02-27 2022-07-06 The Trustees of The University of Pennsylvania NOVEL VECTORS OF ADENO-ASSOCIATED VIRUS (AAV), AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF
MX2020008932A (es) 2018-02-27 2020-10-01 Univ Pennsylvania Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos.
EP3765624A4 (en) * 2018-03-16 2022-05-25 Research Institute at Nationwide Children's Hospital Increasing tissue specific gene delivery by capsid modification
WO2019195449A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
EP3775266A4 (en) 2018-04-05 2021-06-30 Massachusetts Eye and Ear Infirmary METHOD OF MANUFACTURING AND USING COMBINATIONAL BAR-CODED NUCLEIC ACID LIBRARIES WITH DEFINED VARIATION
WO2019193119A1 (en) 2018-04-05 2019-10-10 Genethon HYBRID RECOMBINANT ADENO-ASSOCIATED VIRUS SEROTYPE BETWEEN AAV9 AND AAVrh74 WITH REDUCED LIVER TROPISM
EP3780952A4 (en) 2018-04-16 2022-03-09 The Trustees of the University of Pennsylvania COMPOSITIONS AND METHODS OF TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
AU2019255745A1 (en) 2018-04-18 2020-11-19 The Trustees Of Columbia University In The City Of New York Gene therapy for diseases caused by unbalanced nucleotide pools including mitochondrial DNA depletion syndromes
BR112020021903A2 (pt) 2018-04-27 2021-03-02 Rocket Pharmaceuticals, Ltd. terapia genética para degeneração do snc
CA3098566A1 (en) 2018-04-29 2019-11-07 Zhuchun WU Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles
EP3787771A1 (en) 2018-04-29 2021-03-10 REGENXBIO Inc. Scalable clarification process for recombinant aav production
KR102877876B1 (ko) 2018-05-08 2025-10-28 루트거스, 더 스테이트 유니버시티 오브 뉴 저지 Aav-상용성 라미닌-링커 중합 단백질
WO2019217513A2 (en) 2018-05-09 2019-11-14 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
CN112368391A (zh) 2018-05-11 2021-02-12 马萨诸塞眼科耳科诊所 腺相关病毒的肝脏特异性嗜性
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
TW202015742A (zh) 2018-05-15 2020-05-01 美商航海家醫療公司 投遞腺相關病毒(aav)之組成物和方法
EP3813845A4 (en) 2018-05-15 2022-10-26 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties
MX2020012077A (es) 2018-05-15 2021-03-09 Voyager Therapeutics Inc Composiciones y metodos para el tratamiento de la enfermedad de parkinson.
TWI869346B (zh) 2018-05-30 2025-01-11 瑞士商諾華公司 Entpd2抗體、組合療法、及使用該等抗體和組合療法之方法
US11505782B2 (en) 2018-06-04 2022-11-22 Calidi Biotherapeutics, Inc. Cell-based vehicles for potentiation of viral therapy
ES2966045T3 (es) 2018-06-04 2024-04-18 Calidi Biotherapeutics Inc Vehículos basados en células para la potenciación de la terapia viral
EP3802829A4 (en) 2018-06-08 2022-10-19 University of Massachusetts ANTISENSE OLIGONUCLEOTIDES TO RESTORE DYSFERLIN PROTEIN EXPRESSION IN CELLS FROM PATIENTS WITH DYSFERLINOPATHY
WO2019241315A1 (en) 2018-06-12 2019-12-19 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
WO2019241535A2 (en) 2018-06-14 2019-12-19 Regenxbio Inc. Anion exchange chromatography for recombinant aav production
WO2019241758A1 (en) 2018-06-15 2019-12-19 Alpine Immune Sciences, Inc. Pd-1 variant immunomodulatory proteins and uses thereof
GB201811368D0 (en) 2018-07-11 2018-08-29 Ucb Biopharma Sprl Antibody
CA3106010A1 (en) 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
WO2020016655A2 (en) 2018-07-17 2020-01-23 Helixmith Co., Ltd. Treatment of neuropathy with dna constructs expressing igf-1 isoforms
US20210322473A1 (en) * 2018-07-18 2021-10-21 The General Hospital Corporation Modified t cells and methods of their use
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
WO2020028751A2 (en) 2018-08-03 2020-02-06 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
KR20210043580A (ko) 2018-08-10 2021-04-21 리젠엑스바이오 인크. 재조합 aav 생산을 위한 규모 조정 가능한 방법
WO2020037303A1 (en) 2018-08-16 2020-02-20 The Regents Of The University Of California Chemically and photochemically initiated cell membrane blebbing to induce cell vesicle production, modifications thereof, and uses thereof
WO2020047161A2 (en) 2018-08-28 2020-03-05 Actym Therapeutics, Inc. Engineered immunostimulatory bacterial strains and uses thereof
AU2019333315B2 (en) 2018-08-30 2022-09-29 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with myocardin and ASCL1
KR20210084459A (ko) 2018-09-26 2021-07-07 캘리포니아 인스티튜트 오브 테크놀로지 표적화된 유전자 요법을 위한 아데노-연관 바이러스 조성물
SG11202103151RA (en) 2018-09-28 2021-04-29 Voyager Therapeutics Inc Frataxin expression constructs having engineered promoters and methods of use thereof
JP7289033B2 (ja) 2018-10-01 2023-06-09 徹 宮崎 神経変性疾患治療剤
KR20210071017A (ko) 2018-10-01 2021-06-15 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Gm1 강글리오시드증 치료에 유용한 조성물
CN112955557A (zh) 2018-10-01 2021-06-11 奥特吉尼克斯制药公司 用于治疗丙酸血症的基因疗法
AU2019354995B2 (en) 2018-10-02 2024-12-12 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
EP3861113A1 (en) 2018-10-04 2021-08-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
EP3861107A1 (en) * 2018-10-05 2021-08-11 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
JP2022505106A (ja) 2018-10-15 2022-01-14 ボイジャー セラピューティクス インコーポレイテッド バキュロウイルス/Sf9システムにおけるrAAVの大規模産生のための発現ベクター
EP3867412A1 (en) 2018-10-15 2021-08-25 REGENXBIO Inc. Method for measuring the infectivity of replication defective viral vectors and viruses
MX2021004602A (es) 2018-10-22 2021-09-08 Univ Rochester Edición del genoma por inserción no homóloga de adn dirigida utilizando una proteína de fusión retroviral integrasacas9.
WO2020086742A1 (en) 2018-10-24 2020-04-30 Obsidian Therapeutics, Inc. Er tunable protein regulation
AU2019377141B2 (en) 2018-11-06 2024-06-06 Calidi Biotherapeutics (Nevada), Inc. Enhanced systems for cell-mediated oncolytic viral therapy
BR112021009481A2 (pt) 2018-11-16 2021-08-17 Astellas Pharma Inc. método para tratamento da distrofia muscular direcionando gene de utrofina.
CN113891934A (zh) 2018-11-21 2022-01-04 因达普塔治疗公司 扩增天然杀伤(nk)细胞子集的方法以及相关组合物和方法
BR112021009739A2 (pt) * 2018-11-30 2021-10-19 Novartis Ag Vetores virais aav e usos dos mesmos
CN113727998A (zh) 2018-11-30 2021-11-30 高山免疫科学股份有限公司 Cd86变体免疫调节蛋白及其用途
JP7590968B2 (ja) 2018-12-05 2024-11-27 アビオナ・セラピュ―ティクス・インコーポレイテッド 遺伝子送達のための組み換えアデノ随伴ウイルスベクター
US20220089670A1 (en) 2018-12-28 2022-03-24 University Of Rochester Gene Therapy for BEST1 Dominant Mutations
WO2020142653A1 (en) 2019-01-04 2020-07-09 Ultragenyx Pharmaceutical Inc. Gene therapy constructs for treating wilson disease
JP7416451B2 (ja) 2019-01-14 2024-01-17 ユニバーシティ オブ ロチェスター CRISPR-Casによる標的化された核内RNA切断及びポリアデニル化
CN113631225A (zh) 2019-01-18 2021-11-09 沃雅戈治疗公司 用于生产aav颗粒的方法和系统
US20230193315A1 (en) 2019-01-31 2023-06-22 Oregon Health & Science University Methods for using transcription-dependent directed evolution of aav capsids
EP3927381A1 (en) 2019-02-22 2021-12-29 The Trustees of The University of Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
WO2020174368A1 (en) 2019-02-25 2020-09-03 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
MX2021010266A (es) 2019-02-26 2021-09-23 Univ Pennsylvania Composiciones utiles en el tratamiento de la enfermedad de krabbe.
CA3176660A1 (en) 2019-02-27 2020-09-03 Actym Therapeutics, Inc. Immunostimulatory bacteria engineered to colonize tumors, tumor-resident immune cells, and the tumor microenvironment
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
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
CA3130196A1 (en) 2019-03-25 2020-10-01 Isabelle Richard Production of large-sized quasidystrophins using overlapping aav vectors
SG11202110607WA (en) 2019-04-01 2021-10-28 Tenaya Therapeutics Inc Adeno-associated virus with engineered capsid
CN113966236A (zh) 2019-04-03 2022-01-21 再生生物股份有限公司 眼睛病状的基因疗法
TW202102526A (zh) 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
EP4310495A3 (en) 2019-04-11 2024-04-24 REGENXBIO Inc. Methods of size exclusion chromatography for the characterization of recombinant adeno-associated virus compositions
JP7630443B2 (ja) 2019-04-19 2025-02-17 レジェンクスバイオ インコーポレーテッド アデノ随伴ウイルスベクター製剤及び方法
BR112021021156A2 (pt) 2019-04-24 2022-02-08 Regenxbio Inc Terapêuticos anticorpos totalmente humanos pós-tradução modificados
WO2020223280A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
US20220243225A1 (en) 2019-04-29 2022-08-04 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
PH12021552608A1 (en) * 2019-04-29 2022-06-20 Univ Pennsylvania Novel aav capsids and compositions containing same
WO2020227166A1 (en) 2019-05-03 2020-11-12 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
EP3966227A1 (en) 2019-05-07 2022-03-16 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
AR118928A1 (es) 2019-05-14 2021-11-10 Biomarin Pharm Inc Métodos de redosificación de vectores de terapia génica
CA3135271A1 (en) 2019-05-28 2020-12-03 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting dmpk gene
CN114207120A (zh) 2019-05-30 2022-03-18 坚固生物科技公司 重组疱疹病毒载体
US20230241248A1 (en) 2019-06-27 2023-08-03 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
EP3994253A1 (en) 2019-07-02 2022-05-11 M6P Therapeutics (Switzerland) LLC Vector compositions and methods of using same for treatment of lysosomal storage disorders
WO2021005223A1 (en) 2019-07-10 2021-01-14 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of epilepsy
US20220282276A1 (en) 2019-07-11 2022-09-08 Centre National De La Recherche Scientifique Chemically-modified adeno-associated virus
US20230137971A1 (en) 2019-07-11 2023-05-04 Tenaya Therapeutics Inc. Cardiac cell reprogramming with micrornas and other factors
US10557149B1 (en) 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
US10801042B1 (en) 2019-07-15 2020-10-13 Vigene Biosciences, Inc. Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
JP2022544015A (ja) 2019-07-23 2022-10-17 ユニバーシティ オブ ロチェスター CRISPR-Casでの標的化されたRNA切断
US20230042103A1 (en) 2019-07-26 2023-02-09 Regenxbio Inc. Engineered nucleic acid regulatory element and methods of uses thereof
US20220396806A1 (en) 2019-07-26 2022-12-15 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
WO2021030125A1 (en) 2019-08-09 2021-02-18 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
TW202112797A (zh) 2019-08-16 2021-04-01 日商摩大力斯醫療公司 經由靶向lama1基因治療肌肉萎縮症的方法
US20220288235A1 (en) * 2019-08-20 2022-09-15 St. Jude Children's Research Hospital, Inc. SCHIZOPHRENIA-RELATED MICRODELETION GENE 2510002D24Rik IS ESSENTIAL FOR SOCIAL MEMORY
WO2021041373A1 (en) 2019-08-26 2021-03-04 Regenxbio Inc. Treatment of diabetic retinopathy with fully-human post-translationally modified anti-vegf fab
US20230092895A1 (en) 2019-08-30 2023-03-23 Obsidian Therapeutics, Inc. Tandem cd19 car-based compositions and methods for immunotherapy
EP4021934A4 (en) 2019-08-30 2024-02-14 The Regents of the University of California Gene fragment overexpression screening methodologies, and uses thereof
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
US20220348937A1 (en) 2019-09-06 2022-11-03 Obsidian Therapeutics, Inc. Compositions and methods for dhfr tunable protein regulation
EP4028532A1 (en) 2019-09-13 2022-07-20 Rutgers, the State University of New Jersey Aav-compatible laminin-linker polymerization proteins
US20220348651A1 (en) 2019-09-18 2022-11-03 Novartis Ag Entpd2 antibodies, combination therapies, and methods of using the antibodies and combination therapies
TW202124446A (zh) 2019-09-18 2021-07-01 瑞士商諾華公司 與entpd2抗體之組合療法
CN114521143B (zh) 2019-09-19 2025-03-14 吉尼松公司 减轻fkrp心脏毒性的基因治疗表达系统
JP7620988B2 (ja) 2019-09-20 2025-01-24 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. 血清および組織ライセートからのranタンパク質に対する抗体の検出
WO2021067598A1 (en) 2019-10-04 2021-04-08 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
CN114728049A (zh) 2019-10-07 2022-07-08 再生生物股份有限公司 腺相关病毒载体药物组合物和方法
US20220403438A1 (en) 2019-10-08 2022-12-22 Trustees Of Boston College Proteins containing multiple, different unnatural amino acids and methods of making and using such proteins
JP2022552260A (ja) 2019-10-08 2022-12-15 エクスハウラ リミテッド 眼治療のための組成物および方法
EP4450514A3 (en) 2019-10-14 2025-04-09 Duke University Compositions comprising novel prokaryotic sodium channels and associated methods
TW202128736A (zh) 2019-10-17 2021-08-01 美商史崔德生物公司 用於治療c型尼曼—匹克病之腺相關病毒載體
US20220396808A1 (en) * 2019-11-08 2022-12-15 President And Fellows Of Harvard College Viral capsid polypeptides
EP4055034A4 (en) * 2019-11-08 2024-05-29 President And Fellows Of Harvard College Viral capsid polypeptides
WO2021099394A1 (en) 2019-11-19 2021-05-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Antisense oligonucleotides and their use for the treatment of cancer
KR20220107222A (ko) * 2019-11-28 2022-08-02 리젠엑스바이오 인크. 마이크로디스트로핀 유전자 치료 작제물 및 이의 용도
CN114981442A (zh) * 2019-12-04 2022-08-30 桑格摩生物治疗股份有限公司 用于生产重组aav的新颖组合物及方法
TW202129002A (zh) 2019-12-09 2021-08-01 英商Ucl商業有限責任公司 用於myh7關聯之心肌病之基因療法組合物及治療
EP4072595A1 (en) 2019-12-10 2022-10-19 Takeda Pharmaceutical Company Limited Adeno associated virus vectors for the treatment of hunter disease
AU2020416291A1 (en) 2019-12-31 2022-07-21 Swanbio Therapeutics Limited Improved AAV-ABCD1 constructs and use for treatment or prevention of adrenoleukodystrophy (ALD) and/or adrenomyeloneuropathy (AMN)
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
JP2023511382A (ja) 2020-01-22 2023-03-17 レジェンクスバイオ インコーポレーテッド 完全ヒトグリコシル化ヒトα-L-イズロニダーゼ(IDUA)を用いたムコ多糖症I型の治療
EP4097238A1 (en) 2020-01-29 2022-12-07 REGENXBIO Inc. Treatment of mucopolysaccharidosis iva
TW202142695A (zh) 2020-01-29 2021-11-16 美商銳進科斯生物股份有限公司 用人類神經或神經膠質細胞產生之重組人類艾杜糖醛酸—2—硫酸酯酶(ids)對ii型黏多糖病之治療
WO2021155337A1 (en) 2020-02-02 2021-08-05 The Trustees Of The University Of Pennsylvania Compositions useful for treating gm1 gangliosidosis
KR20220141829A (ko) 2020-02-07 2022-10-20 유니버시티 오브 로체스터 리보자임-매개 rna 조립 및 발현
JP2023513211A (ja) 2020-02-07 2023-03-30 ユニバーシティ オブ ロチェスター タンパク質合成を増強するためのCRISPR-Cas13による標的RNA翻訳
IL294781A (en) 2020-02-14 2022-09-01 Ultragenyx Pharmaceutical Inc Gene therapy for treating cdkl5 deficiency disorder
WO2021170784A1 (en) 2020-02-28 2021-09-02 INSERM (Institut National de la Santé et de la Recherche Médicale) Gene therapy for maple syrup urine disease
CA3173567A1 (en) 2020-03-02 2021-09-10 Tenaya Therapeutics, Inc. Gene vector control by cardiomyocyte-expressed micrornas
MX2022010936A (es) 2020-03-05 2022-11-16 Neotx Therapeutics Ltd ³métodos y composiciones para el tratamiento del cáncer con células inmunológicas.
EP4118098A4 (en) 2020-03-11 2024-06-12 The Trustees of The University of Pennsylvania METHOD AND COMPOSITION FOR GENE DELIVERY USING AN MANIPULATED VIRUS PARTICLE
EP4121544A1 (en) * 2020-03-19 2023-01-25 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
WO2021195519A1 (en) 2020-03-27 2021-09-30 University Of Rochester Targeted destruction of viral rna by crispr-cas13
JP2023519344A (ja) 2020-03-27 2023-05-10 ユニバーシティ オブ ロチェスター CRISPR-Cas13 crRNAアレイ
IL296732A (en) 2020-03-27 2022-11-01 UCB Biopharma SRL Autonomous knob domain peptides
EP4127189A1 (en) 2020-03-31 2023-02-08 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
WO2021202651A1 (en) 2020-04-01 2021-10-07 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
US20230226223A1 (en) 2020-04-10 2023-07-20 Sola Biosciences Llc Compositions and Methods for the Treatment of Protein Aggregation Disorders
MX2022012752A (es) 2020-04-15 2023-01-16 Voyager Therapeutics Inc Compuestos de union a tau.
US20230220014A1 (en) * 2020-04-20 2023-07-13 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
CN116368221A (zh) 2020-04-22 2023-06-30 因达普塔治疗公司 自然杀伤(nk)细胞组合物及其生成方法
KR20230025659A (ko) 2020-04-28 2023-02-22 솔라 바이오사이언시즈 엘엘씨 Tdp-43 단백질병증의 치료를 위한 조성물 및 방법
MX2022013963A (es) 2020-05-05 2023-02-22 Univ Duke Composiciones de virus adenoasociados compatibles entre especies y metodos de uso de las mismas.
TW202208622A (zh) 2020-05-12 2022-03-01 賓州大學委員會 用於治療克拉培氏病之組成物
AU2021270447A1 (en) 2020-05-12 2023-01-05 The Trustees Of The University Of Pennsylvania Compositions for DRG-specific reduction of transgene expression
JP2023526047A (ja) 2020-05-13 2023-06-20 アコーオス インコーポレイテッド Slc26a4関連難聴を治療するための組成物及び方法
US20230203102A1 (en) 2020-05-13 2023-06-29 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
TW202208406A (zh) 2020-05-13 2022-03-01 美商阿科奧斯公司 用於治療kcnq4相關性聽力損失之組成物及方法
WO2021230385A1 (en) 2020-05-15 2021-11-18 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
WO2021236908A2 (en) 2020-05-20 2021-11-25 Biomarin Pharmaceutical Inc. Use of regulatory proteins for the production of adeno-associated virus
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
WO2021248038A1 (en) 2020-06-05 2021-12-09 Sola Biosciences Llc Compositions and methods for the treatment of synucleinopathies
CA3183153A1 (en) 2020-06-17 2021-12-23 Christian HINDERER Compositions and methods for treatment of gene therapy patients
WO2021255245A2 (en) 2020-06-19 2021-12-23 Genethon Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg
CA3187635A1 (en) * 2020-07-03 2022-01-06 Genethon Method for engineering novel hybrid aav capsids through hypervariable regions swapping
KR20230035584A (ko) * 2020-07-07 2023-03-14 더 보드 오브 리젠츠 오브 더 유니버시티 오브 텍사스 시스템 Dwarf 개방 판독 프레임을 위한 아데노-관련 바이러스 벡터
US20230277686A1 (en) 2020-07-10 2023-09-07 Genethon A novel muscle-specific promoter
IL299771A (en) 2020-07-10 2023-03-01 Inst Nat Sante Rech Med Methods and compounds for the treatment of epilepsy
TW202216244A (zh) 2020-07-13 2022-05-01 賓州大學委員會 有用於治療夏馬杜三氏病之組成物
US20230348871A1 (en) 2020-07-15 2023-11-02 University Of Rochester Targeted RNA cleavage with dCas13-RNase Fusion Proteins
CA3190309A1 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
CN116121275B (zh) * 2020-07-29 2024-11-29 北京三诺佳邑生物技术有限责任公司 一组肝靶向新型腺相关病毒的获得及其应用
US20230295656A1 (en) 2020-08-06 2023-09-21 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
EP4196494A1 (en) 2020-08-17 2023-06-21 Massachusetts Institute of Technology Shank3 gene therapy approaches
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
MX2023002293A (es) 2020-08-24 2023-05-19 Univ Pennsylvania Vectores virales que codifican fusiones de agonistas del receptor de glp-1 y usos de estos en el tratamiento de enfermedades metabólica.
MX2023002364A (es) 2020-08-26 2023-05-22 Univ Pennsylvania Virus adenoasociado recombinante para el tratamiento de la neurodegeneración iniciada en adultos asociada a grn.
BR112023003972A2 (pt) 2020-09-04 2023-04-18 Univ Kobe Nat Univ Corp Complexos de um módulo de reconhecimento de sequência de ácido nucleico ligado a uma desaminase e de um fragmento amino-terminal de um módulo de reconhecimento de sequência de ácido nucleico, uma desaminase e um fragmento carboxi-terminal de um módulo de reconhecimento de sequência de ácido nucleico ligado ao mesmo, ácido nucleico, vetor, e, método para alterar um sítio alvo de um ácido desoxirribonucleico de fita dupla de uma célula
WO2022060916A1 (en) 2020-09-15 2022-03-24 Regenxbio Inc. Vectorized antibodies for anti-viral therapy
EP4213890A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized lanadelumab and administration thereof
US20230383278A1 (en) 2020-09-18 2023-11-30 The United States Of America,As Represented By The Secretary,Department Of Health And Human Services Novel adeno-associated viral (aav) vectors to treat hereditary methylmalonic acidemia (mma) caused by methylmalonyl-coa mutase (mmut) deficiency
AU2021358964A1 (en) 2020-10-07 2023-05-25 Regenxbio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
WO2022076549A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations for suprachoroidal administration such as high viscosity formulations
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
IL301647A (en) 2020-10-07 2023-05-01 Regenxbio Inc Adeno-associated viruses for ocular delivery of gene therapy
JP2023544803A (ja) 2020-10-07 2023-10-25 レジェンクスバイオ インコーポレーテッド Cln2疾患の眼症状に対する遺伝子療法
US20230414788A1 (en) 2020-10-07 2023-12-28 Regenxbio Inc. Formulations for suprachoroidal administration such as gel formulations
KR20230118075A (ko) 2020-10-09 2023-08-10 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 파브리병 치료를 위한 조성물 및 방법
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
TW202229315A (zh) 2020-10-18 2022-08-01 賓州大學委員會 改良的腺相關病毒(aav)載體及其用途
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
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
EP4236974A2 (en) 2020-10-29 2023-09-06 RegenxBio Inc. Vectorized factor xii antibodies and administration thereof
CN116457373A (zh) 2020-10-29 2023-07-18 再生生物股份有限公司 用于眼部适应症的载体化TNF-α拮抗剂
WO2022094461A1 (en) 2020-11-02 2022-05-05 Biomarin Pharmaceutical Inc. Process for enriching adeno-associated virus
IL302608A (en) 2020-11-03 2023-07-01 Pfizer Methods for purification of aav vectors by anion exchange chromatography
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
EP4256065A2 (en) 2020-12-01 2023-10-11 The Trustees of The University of Pennsylvania Novel compositions with tissue-specific targeting motifs and compositions containing same
CA3200192A1 (en) 2020-12-01 2022-06-09 Justin PERCIVAL Compositions and uses thereof for treatment of angelman syndrome
US20240052322A1 (en) 2020-12-15 2024-02-15 Pfizer Inc. HILIC UPLC-MS Method For Separating and Analyzing Intact Adeno-Associated Virus Capsid Proteins
CA3201743A1 (en) 2020-12-16 2022-06-23 Robert STADELMAN Method of producing a recombinant adeno-associated virus particle
IL303959A (en) 2020-12-23 2023-08-01 Pfizer Methods for purification of aav vectors by affinity chromatography
EP4271419A1 (en) 2020-12-29 2023-11-08 Akouos, Inc. Compositions and methods for treating clrn1-associated hearing loss and/or vision loss
WO2022147087A1 (en) 2020-12-29 2022-07-07 Regenxbio Inc. Tau-specific antibody gene therapy compositions, methods and uses thereof
WO2022147481A1 (en) 2020-12-30 2022-07-07 Ansun Biopharma Inc. Combination therapy of an oncolytic virus delivering a foreign antigen and an engineered immune cell expressing a chimeric receptor targeting the foreign antigen
CN116981697A (zh) 2021-01-14 2023-10-31 森迪生物科学公司 可分泌有效载荷调节
JP2024503895A (ja) 2021-01-21 2024-01-29 リジェネクスバイオ インコーポレイテッド 組換えポリペプチド及びウイルスの生成の改善
KR20230149299A (ko) * 2021-01-25 2023-10-26 트라메스 바이오 인코포레이티드 초점 간질 및 신경병성 통증 치료를 위한 아데노-연관 바이러스 캡시드 및 조작된 리간드 개폐 이온 채널
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
US11926842B2 (en) * 2021-02-02 2024-03-12 University Of Massachusetts Use of inverted terminal repeats (ITRS) from adeno-associated virus serotypes 8 and RH.39 in gene therapy vectors
JP2024506296A (ja) 2021-02-05 2024-02-13 メイズ セラピューティクス, インコーポレイテッド スタッファーポリヌクレオチド配列を含むベクター
AU2022218706A1 (en) * 2021-02-09 2023-09-28 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
CA3209611A1 (en) 2021-02-10 2022-08-18 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
AR124981A1 (es) 2021-02-26 2023-05-24 Takeda Pharmaceuticals Co Composición y métodos para el tratamiento de la enfermedad de fabry
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
JP2024511024A (ja) 2021-03-19 2024-03-12 アドリーナス セラピューティクス, インコーポレイテッド 21-ヒドロキシラーゼ欠損症の遺伝子治療
WO2022208342A1 (en) 2021-04-01 2022-10-06 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
WO2022216749A1 (en) 2021-04-05 2022-10-13 Solid Biosciences Inc. Recombinant herpesvirales vector
US20240191258A1 (en) 2021-04-12 2024-06-13 The Trustees Of The University Of Pennsylvania Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
CA3216146A1 (en) 2021-04-23 2022-10-27 Douglas Anderson Genome editing by directed non-homologous dna insertion using a retroviral integrase-cas fusion protein and methods of treatment
EP4334334A1 (en) 2021-04-23 2024-03-13 The Trustees of The University of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
WO2022229807A1 (en) 2021-04-26 2022-11-03 Pfizer Inc. Adeno-associated viral vector capsids with improved tissue tropism
CA3216744A1 (en) 2021-04-26 2022-11-03 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies
US20240218397A1 (en) 2021-05-04 2024-07-04 Regenxbio Inc. Novel aav vectors and methods and uses thereof
US20240358857A1 (en) 2021-05-11 2024-10-31 Regenxbio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
JP2024520815A (ja) * 2021-06-08 2024-05-24 エヌエフ2 セラピューティクス, インコーポレイテッド 神経線維腫性障害を処置するための組成物および方法
EP4355888A4 (en) * 2021-06-18 2025-07-02 Dyno Therapeutics Inc CAPSID VARIANTS AND METHODS OF USE THEREOF
US20240271160A1 (en) 2021-06-22 2024-08-15 Pfizer Inc. Production of adeno-associated virus vector in insect cells
US20240287493A1 (en) * 2021-06-24 2024-08-29 University Of Utah Research Foundation Compositions and methods for treating pgm1 deficiency
EP4362957A1 (en) 2021-07-01 2024-05-08 Indapta Therapeutics, Inc. Engineered natural killer (nk) cells and related methods
AU2022307365A1 (en) 2021-07-08 2024-02-01 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
EP4373947A1 (en) 2021-07-19 2024-05-29 New York University Auf1 combination therapies for treatment of muscle degenerative disease
CN118076744A (zh) * 2021-07-23 2024-05-24 杜克大学 腺相关病毒组合物及其使用方法
IL310515A (en) 2021-07-30 2024-03-01 Toru Miyazaki Therapeutic agent for ischemic disease
WO2023019225A2 (en) 2021-08-11 2023-02-16 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce instant blood mediated inflammatory reactions
EP4384544A1 (en) 2021-08-11 2024-06-19 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy
AU2022325232A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified primary cells for allogeneic cell therapy
EP4384598A1 (en) 2021-08-11 2024-06-19 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions
TW202338095A (zh) * 2021-08-25 2023-10-01 美商北海康成製藥有限公司 包含肝臟向性衣殼蛋白質及酸α-葡萄糖苷酶(GAA)之AAV顆粒及其用於治療龐貝氏病(POMPE DISEASE)之用途
KR20240073025A (ko) * 2021-08-25 2024-05-24 캔브리지 파마슈티컬스, 인크. 간-향성 캡시드 단백질 및 알파-갈락토시다제를 포함하는 aav 입자 및 파브리 질환을 치료하기 위한 이의 용도
KR20240107201A (ko) 2021-08-31 2024-07-08 스카우트 바이오, 인코포레이티드 항원-결합 분자 및 이의 용도
EP4405396A2 (en) 2021-09-20 2024-07-31 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
IL311572A (en) 2021-09-30 2024-05-01 Akouos Inc Compositions and methods for treating kcnq4-associated hearing loss
TW202325845A (zh) 2021-10-02 2023-07-01 賓州大學委員會 新穎aav衣殼及含其之組成物
CN118202060A (zh) 2021-10-05 2024-06-14 再生生物股份有限公司 用于重组aav生产的组合物和方法
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2023060269A1 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
US20230151372A1 (en) * 2021-10-07 2023-05-18 Academia Sinica Composition and Method of Treatment for Heart Protection and Regeneration
WO2023060272A2 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
MX2024004217A (es) 2021-10-08 2024-06-26 Dyno Therapeutics Inc Variantes de cápside y métodos de uso de estas.
CA3234720A1 (en) 2021-10-08 2023-04-13 Sola Biosciences Llc Compositions and methods for the treatment of p53-mediated cancers
WO2023060221A2 (en) 2021-10-08 2023-04-13 Sola Biosciences Llc Compositions and methods for the treatment of proteopathies
EP4415746A1 (en) 2021-10-12 2024-08-21 Bridgebio Gene Therapy LLC Methods and compositions for treating leukodystrophies
US20230241118A1 (en) 2021-10-20 2023-08-03 University Of Rochester Rejuvenation treatment of age-related white matter loss
EP4423285A1 (en) 2021-10-28 2024-09-04 RegenxBio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
CA3236365A1 (en) 2021-11-02 2023-05-11 University Of Rochester Tcf7l2 mediated remyelination in the brain
KR20240113624A (ko) 2021-11-02 2024-07-22 보이저 테라퓨틱스, 인크. Aav 캡시드 변이체 및 이의 용도
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
US20250034559A1 (en) 2021-11-17 2025-01-30 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
EP4433169A1 (en) 2021-11-17 2024-09-25 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
WO2023093905A1 (zh) 2021-11-29 2023-06-01 上海瑞宏迪医药有限公司 Aadc、gdnf多核苷酸及其用于治疗帕金森病
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
WO2023114897A2 (en) * 2021-12-15 2023-06-22 Homology Medicines, Inc. Methods and compositions for the production of adeno-associated virus
EP4463135A2 (en) 2022-01-10 2024-11-20 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
TW202340467A (zh) 2022-01-10 2023-10-16 賓州大學委員會 有用於治療c9orf72介導之病症之組成物及方法
WO2023133584A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
CA3248878A1 (en) 2022-01-17 2023-07-20 Dinaqor Ag GENE THERAPY COMPOSITION AND TREATMENT OF DYSTROPHIN-RELATED CARDIOMYOPATHY
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
IL314434A (en) 2022-01-25 2024-09-01 Univ Pennsylvania AAV capsids for enhanced cardiac transduction and liver targeting neutralization
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
AU2023215253A1 (en) 2022-02-02 2024-09-19 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
US20250144235A1 (en) 2022-02-02 2025-05-08 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
AU2023217698A1 (en) 2022-02-08 2024-07-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
KR20240155390A (ko) 2022-02-17 2024-10-28 사나 바이오테크놀로지, 인크. 조작된 cd47 단백질 및 이의 용도
WO2023155918A1 (zh) 2022-02-21 2023-08-24 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
EP4482970A1 (en) 2022-02-23 2025-01-01 Massachusetts Institute of Technology Methods for upregulating shank3 expression
US20250101382A1 (en) 2022-03-11 2025-03-27 Sana Biotechnology, Inc. Genetically modified cells and compositions and uses thereof
EP4493226A1 (en) 2022-03-13 2025-01-22 RegenxBio Inc. Modified muscle-specific promoters
EP4499154A1 (en) 2022-03-25 2025-02-05 REGENXBIO Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
WO2023187728A1 (en) 2022-04-01 2023-10-05 Takeda Pharmaceutical Company Limited Gene therapy for diseases with cns manifestations
AU2023250660A1 (en) 2022-04-06 2024-10-24 Regenxbio Inc. Pharmaceutical composition comprising a recombinant adenoassociated virus vector with an expression cassette encoding a transgene for suprachoroidal administration
EP4504258A1 (en) 2022-04-06 2025-02-12 The Trustees of the University of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
CA3247109A1 (en) 2022-04-06 2023-10-12 Univ Pennsylvania Compositions and treatment methods for metastatic breast cancer that is human epidermal growth factor receptor 2 positive and other cancers
TW202345913A (zh) 2022-04-06 2023-12-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如凝膠調配物
TW202404651A (zh) 2022-04-06 2024-02-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如形成聚集體之調配物
JP2025513835A (ja) 2022-04-11 2025-04-30 サントル ナショナル ドゥ ラ ルシェルシュ シアンティフィック 化学修飾型アデノ随伴ウイルス
WO2023201207A1 (en) 2022-04-11 2023-10-19 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
US20250249127A1 (en) 2022-04-14 2025-08-07 Regenxbio Inc. Gene therapy for treating an ocular disease
WO2023201277A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
WO2023205610A2 (en) 2022-04-18 2023-10-26 Regenxbio Inc. Hybrid aav capsids
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
EP4518972A2 (en) 2022-05-03 2025-03-12 RegenxBio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
US20250288697A1 (en) 2022-05-03 2025-09-18 Regenxbio Inc. Vectorized anti-tnf-alpha inhibitors for ocular indications
US20250353883A1 (en) 2022-05-06 2025-11-20 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
EP4522757A2 (en) 2022-05-13 2025-03-19 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
IL317433A (en) * 2022-05-24 2025-02-01 Kate Therapeutics Inc Compounds for the treatment of XLMTM
CA3257081A1 (en) 2022-06-02 2023-12-07 Voyager Therapeutics Inc VAA CAPSIDE VARIANTS AND THEIR USES
WO2023240158A2 (en) * 2022-06-07 2023-12-14 Oregon Health & Science University Mutant adeno-associated virus (aav) capsids
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
WO2023250511A2 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
WO2024006741A1 (en) 2022-06-28 2024-01-04 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
CA3259982A1 (en) 2022-06-30 2024-01-04 Indapta Therapeutics Inc COMBINATION OF MODIFIED NATURAL KILLER (NK) CELLS AND ANTIBODY THERAPY AND ASSOCIATED METHODS
WO2024011112A1 (en) 2022-07-06 2024-01-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
EP4558149A1 (en) 2022-07-21 2025-05-28 Institut National de la Santé et de la Recherche Médicale Methods and compositions for treating chronic pain disorders
WO2024026377A1 (en) 2022-07-27 2024-02-01 Sana Biotechnology, Inc. Methods of transduction using a viral vector and inhibitors of antiviral restriction factors
TW202435912A (zh) 2022-08-03 2024-09-16 美商航海家醫療公司 用於穿過血腦屏障之組合物及方法
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
EP4577663A2 (en) 2022-08-24 2025-07-02 RegenxBio Inc. Recombinant adeno-associated viruses and uses thereof
CN119768188A (zh) 2022-08-25 2025-04-04 武田药品工业株式会社 用于治疗法布里病的组合物
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2024064863A2 (en) 2022-09-22 2024-03-28 Biomarin Pharmaceutical Inc. Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors
UY40443A (es) 2022-09-22 2024-02-15 Biomarin Pharm Inc Tratamiento de la miocardiopatía con vectores de genoterapia con aav
IL319873A (en) 2022-09-30 2025-05-01 Regenxbio Inc Treatment of eye diseases with recombinant viral vectors containing anti-VEGF FAB
WO2024077089A2 (en) * 2022-10-04 2024-04-11 Siren Biotechnology, Inc. Modified cpg dinucleotides for recombinant viral vector production
JP2025534666A (ja) 2022-10-11 2025-10-17 リジェネックスバイオ インコーポレイテッド 操作された核酸調節エレメントならびにその使用方法
WO2024105638A1 (en) 2022-11-18 2024-05-23 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of hunter syndrome
CN121127599A (zh) 2022-12-17 2025-12-12 宾夕法尼亚州大学信托人 具有心肌和骨骼肌特异性靶向基序的重组aav突变载体及含有其的组合物
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
IT202200026595A1 (it) 2022-12-22 2024-06-22 Fond Telethon Ets Nuovi inibitori di regolatori epigenetici
CN120752339A (zh) 2023-01-06 2025-10-03 国家医疗保健研究所 静脉内施用用于治疗疼痛的反义寡核苷酸
WO2024151541A1 (en) 2023-01-09 2024-07-18 Sana Biotechnology, Inc. Type-1 diabetes autoimmune mouse
EP4649160A1 (en) 2023-01-12 2025-11-19 Nantes Université Chemically-modified adeno-associated virus
WO2024163979A2 (en) * 2023-02-02 2024-08-08 Codexis, Inc. Engineered aav polypeptides
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
EP4658294A2 (en) 2023-02-02 2025-12-10 University of Rochester Competitive replacement of glial cells
AU2024221161A1 (en) 2023-02-17 2025-08-28 Biogen Ma Inc. Rgd-containing peptides for delivering payloads
WO2024192281A2 (en) 2023-03-15 2024-09-19 Regenxbio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
KR20250158036A (ko) 2023-03-17 2025-11-05 유니버시티 오브 로체스터 리보자임-매개 rna 조립 및 발현
WO2024206226A1 (en) * 2023-03-25 2024-10-03 Duke University Compositions comprising kidney-tropic aavs and methods of use thereof
WO2024211780A1 (en) 2023-04-07 2024-10-10 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
WO2024220463A2 (en) * 2023-04-17 2024-10-24 University Of Massachusetts Aav5 capsid with non-canonical amino acid incorporation and uses thereof
WO2024220389A2 (en) * 2023-04-17 2024-10-24 University Of Massachusetts Aav2 variants and uses thereof
AU2024258047A1 (en) 2023-04-18 2025-10-23 Uniqure Biopharma B.V. Novel neurotropic adeno-associated virus capsids with detargeting of peripheral organs
WO2024218190A1 (en) 2023-04-18 2024-10-24 Uniqure Biopharma B.V. Generation of adeno-associated virus capsid libraries for insect cells
TW202449167A (zh) 2023-04-26 2024-12-16 美商航海家醫療公司 用於治療肌肉萎縮性脊髓側索硬化症之組成物及方法
WO2024226790A1 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024229389A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2024229125A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to frataxin deficiency
AU2024264889A1 (en) 2023-05-03 2025-11-13 Sana Biotechnology, Inc. Methods of dosing and administration of engineered islet cells
AU2024266138A1 (en) 2023-05-03 2025-11-13 Manifold Biotechnologies, Inc. Methods and compositions for high-throughput protein delivery, screening, and detection
CN121079310A (zh) 2023-05-03 2025-12-05 沃雅戈治疗公司 用于治疗与cdkl5缺乏相关的病症的组合物及方法
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
WO2024229425A1 (en) 2023-05-04 2024-11-07 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024233422A1 (en) 2023-05-05 2024-11-14 Massachusetts Institute Of Technology Shank3 gene therapy approaches
WO2024233529A2 (en) 2023-05-07 2024-11-14 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024238867A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
WO2024238853A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof
WO2024243236A2 (en) 2023-05-22 2024-11-28 Sana Biotechnology, Inc. Methods of delivery of islet cells and related methods
WO2024241176A1 (en) 2023-05-24 2024-11-28 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of diseases with central nervous system disorders
WO2024258961A1 (en) 2023-06-12 2024-12-19 The Trustees Of The University Of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
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
WO2025008406A1 (en) 2023-07-04 2025-01-09 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of cancer
WO2025017168A1 (en) 2023-07-19 2025-01-23 Genethon Novel optimized utrophin micro-genes
WO2025017169A1 (en) 2023-07-20 2025-01-23 Genethon Novel mididystrophins
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025042711A1 (en) 2023-08-18 2025-02-27 Eli Lilly And Company Engineered transferrin receptor binding peptides as well as methods of making and using the same
WO2025072604A1 (en) 2023-09-28 2025-04-03 University Of Rochester Rna-editing gene therapy approaches for treating myotonic dystrophy type 1 (dm1)
WO2025075963A1 (en) 2023-10-02 2025-04-10 Regenxbio Inc. Methods and formulations for treating mucopolysaccharidosis ii-associated hearing loss with recombinant human iduronate-2-sulfatase
WO2025080780A1 (en) 2023-10-10 2025-04-17 University Of Rochester Delivery and expression of prime editing crispr systems
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
WO2025090962A1 (en) 2023-10-25 2025-05-01 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2025090858A1 (en) 2023-10-27 2025-05-01 Biogen Ma Inc. Methods for identifying aav capsid variants with desired characteristics
WO2025096967A1 (en) 2023-11-02 2025-05-08 Biogen Ma Inc. Redirection of aav capsids for central nervous system targeting
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
WO2025106374A1 (en) 2023-11-13 2025-05-22 Juno Therapeutics, Inc. Aav production method
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
WO2025108407A2 (en) 2023-11-23 2025-05-30 Neuexcell Therapeutics (Suzhou) Co., Ltd. Gene therapy compositions and methods for treating glioma
WO2025113676A1 (en) 2023-11-29 2025-06-05 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating stroke in primates
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
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
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
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
WO2025171001A1 (en) 2024-02-06 2025-08-14 Regeneron Pharmaceuticals, Inc. Compositions and methods for improved production of aav viral vectors
WO2025188625A1 (en) 2024-03-03 2025-09-12 Passage Bio, Inc. Recombinant adeno-associated virus for treatment of neurodegenerative disorders
WO2025188755A1 (en) 2024-03-04 2025-09-12 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
WO2025207948A1 (en) 2024-03-27 2025-10-02 Biogen Ma Inc. Aav capsids for targeting human transferrin receptor
WO2025207932A1 (en) 2024-03-27 2025-10-02 Biogen Ma Inc. Aav capsids comprising anti-transferrin antibody domains
WO2025217230A1 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
WO2025217214A2 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
WO2025226841A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Gene therapy approach for treating disorders associated with tnnt2
WO2025235734A2 (en) 2024-05-09 2025-11-13 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025237990A1 (en) 2024-05-14 2025-11-20 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of pulmonary fibrosis
GB202407038D0 (en) 2024-05-17 2024-07-03 Axovia Therapeutics Ltd Novel Gene Therapy
WO2025242657A1 (en) 2024-05-21 2025-11-27 Shape Biopharmaceuticals Ag Lipopeptide building blocks and aggregates
WO2025250454A1 (en) 2024-05-28 2025-12-04 University Of Rochester Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo

Family Cites Families (58)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4886876A (en) 1983-03-31 1989-12-12 Scripps Clinic And Research Foundation Factor VIII coagulant polypeptides
JPH07106156B2 (ja) 1983-10-28 1995-11-15 ジェネティックス、インスティチュ−ト ファクタ−▲viii▼および関連生産物の製造
US4757006A (en) 1983-10-28 1988-07-12 Genetics Institute, Inc. Human factor VIII:C gene and recombinant methods for production
US5045455A (en) 1984-01-12 1991-09-03 Chiron Corporation Factor VIII:C cDNA cloning and expression
US4965199A (en) 1984-04-20 1990-10-23 Genentech, Inc. Preparation of functional human factor VIII in mammalian cells using methotrexate based selection
FI86885C (fi) 1984-04-20 1992-10-26 Genentech Inc Foerfarande foer framstaellning av human rekombinantfaktor viii och nukleinsyrasekvenser och vektorer anvaend daertill
EP0182448A3 (en) 1984-08-24 1987-10-28 Genetics Institute, Inc. Production of factor viii and related products
FI98829C (fi) 1986-01-27 1997-08-25 Chiron Corp Menetelmä rekombinoidun proteiinikompleksin valmistamiseksi, jolla on humaanitekijä VIII:C-aktiivisuutta
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US5422260A (en) 1986-05-29 1995-06-06 Genetics Institute, Inc. -Legal Affairs Human factor VIII:c muteins
US5451521A (en) 1986-05-29 1995-09-19 Genetics Institute, Inc. Procoagulant proteins
US5149637A (en) 1987-04-06 1992-09-22 Scripps Clinic & Research Foundation Recombinant Factor VIIIC fragments
IL86693A (en) 1987-06-12 1994-06-24 Stichting Centraal Lab Proteins with factor VIII activity, process for their preparation using genetically engineered cells and pharmaceutical compositions containing them
FR2619314B1 (fr) 1987-08-11 1990-06-15 Transgene Sa Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant
US5004803A (en) 1988-11-14 1991-04-02 Genetics Institute, Inc. Production of procoagulant proteins
FI922204L (fi) 1989-11-17 1992-05-14 Novo Nordisk As Proteinkomplex med faktor viii:c- aktivitet och framstaellning av desamma.
SE465222C5 (sv) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning
US5661008A (en) 1991-03-15 1997-08-26 Kabi Pharmacia Ab Recombinant human factor VIII derivatives
SE468050C (sv) 1991-03-15 1998-04-27 Pharmacia & Upjohn Ab Rekombinant derivat av human faktor VIII
CA2078721A1 (en) 1991-09-24 1993-03-25 Hiroshi Yonemura Process for preparing human coagulation factor viii protein complex
AU5602394A (en) 1992-11-13 1994-06-08 Duke University Chimeric blood coagulation proteins
US5563045A (en) 1992-11-13 1996-10-08 Genetics Institute, Inc. Chimeric procoagulant proteins
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
EP0629700A3 (en) 1993-06-10 1995-02-15 Miles Inc Mammalian vector and cell lines that have increased productivity.
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5681746A (en) 1994-12-30 1997-10-28 Chiron Viagene, Inc. Retroviral delivery of full length factor VIII
WO1997003195A1 (en) 1995-07-11 1997-01-30 Chiron Corporation Novel factor viii:c polypeptide analogs with altered protease sites
US20020088014A1 (en) * 1996-05-31 2002-07-04 Xiangming Fang Minimal adenovirus mediated recombinant vaccine
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
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
ATE465267T1 (de) * 1996-09-06 2010-05-15 Univ Pennsylvania Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen
EP0932694A2 (en) 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
JP2001517454A (ja) 1997-09-19 2001-10-09 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 組換えアデノ随伴ウイルスの産生に有用な方法および細胞株
ES2313784T3 (es) 1998-05-28 2009-03-01 The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services Vector aav5 y usos del mismo.
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
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
US6498244B1 (en) * 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
AU2002248297A1 (en) * 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
CN1236057C (zh) * 2001-10-18 2006-01-11 华中科技大学同济医学院附属同济医院 表达人类激肽释放酶的重组腺相关病毒及其制法和用途
CA2915124C (en) 2001-11-13 2018-08-14 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2602352T3 (es) 2001-12-17 2017-02-20 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas
JP4769417B2 (ja) * 2001-12-17 2011-09-07 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 アデノ随伴ウイルス(aav)血清型9の配列、それを含むベクターおよびその使用
US20060292117A1 (en) * 2002-04-17 2006-12-28 Loiler Scott A Improved rAAv vectors
US7709224B2 (en) * 2003-06-03 2010-05-04 Biosante Pharmaceuticals, Inc. Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site
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
EP2357010B1 (en) * 2005-04-07 2013-06-12 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
US8734809B2 (en) * 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
JP6224459B2 (ja) * 2011-02-17 2017-11-01 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 組織特異性を改変し、aav9媒介遺伝子導入を改善するための組成物および方法
EP2847337A4 (en) 2012-05-09 2016-04-27 Univ Oregon Health & Science ADENO ASSOCIATED VIRUS PLASMIDS AND VECTORS
EP3800260A1 (en) 2014-09-24 2021-04-07 City of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
SG11201907611WA (en) * 2017-02-28 2019-09-27 Univ Pennsylvania Influenza vaccines based on aav vectors
US11827906B2 (en) 2017-02-28 2023-11-28 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clade f vector and uses therefor

Similar Documents

Publication Publication Date Title
JP2014012022A5 (enExample)
FI3313991T3 (fi) Muunnettu tekijä IX sekä koostumukset, menetelmät ja käytöt geeninsiirtoa varten soluihin, elimiin ja kudoksiin
JP2011101646A5 (enExample)
FI3684423T3 (fi) Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä
JP2017532966A5 (enExample)
JP2018520997A5 (enExample)
FI3117005T3 (fi) Hyönteissoluissa tuotettuja lisää parannettuja aav-vektoreita
JP2019533439A5 (enExample)
JP2014239686A5 (enExample)
JP2018108113A5 (enExample)
JP2018528253A5 (enExample)
JP2017512466A5 (enExample)
JP2018515096A5 (enExample)
JP2017536116A5 (enExample)
JP2015096070A5 (enExample)
FI3872085T3 (fi) Esimuotoiset virussekvenssit ja niiden käytöt
JP2020522269A5 (enExample)
PE20160188A1 (es) Variantes aav y composiciones, metodos y usos para transferencia de gen a celulas, organos y tejidos
JP2020520662A5 (enExample)
MX372992B (es) Vector de virus adeno-asociado.
JP2019513794A5 (enExample)
HRP20201324T1 (hr) Vektori aav koji sadrže faktor viii koji kodira gen
JP2018522529A5 (enExample)
JP2015057052A5 (enExample)
RU2014146159A (ru) Композиция и способы высокоэффективного переноса генов с помощью вариантов капсида aav