JP2008538286A5 - - Google Patents

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JP2008538286A5
JP2008538286A5 JP2008505635A JP2008505635A JP2008538286A5 JP 2008538286 A5 JP2008538286 A5 JP 2008538286A5 JP 2008505635 A JP2008505635 A JP 2008505635A JP 2008505635 A JP2008505635 A JP 2008505635A JP 2008538286 A5 JP2008538286 A5 JP 2008538286A5
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amino acid
seq
nature
modified
aav
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JP2008505635A
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JP5702519B2 (ja
JP2008538286A (ja
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Priority claimed from PCT/US2006/013375 external-priority patent/WO2006110689A2/en
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JP2008505635A 2005-04-07 2006-04-07 Aavベクターの機能を上げる方法 Active JP5702519B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US66908305P 2005-04-07 2005-04-07
US60/669,083 2005-04-07
US73349705P 2005-11-04 2005-11-04
US60/733,497 2005-11-04
PCT/US2006/013375 WO2006110689A2 (en) 2005-04-07 2006-04-07 Method of increasing the function of an aav vector

Related Child Applications (2)

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JP2012055665A Division JP5677681B2 (ja) 2005-04-07 2012-03-13 改変されたaavベクター
JP2014244953A Division JP6039632B2 (ja) 2005-04-07 2014-12-03 Aavベクターの機能を上げる方法

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JP2008538286A JP2008538286A (ja) 2008-10-23
JP2008538286A5 true JP2008538286A5 (enExample) 2009-05-28
JP5702519B2 JP5702519B2 (ja) 2015-04-15

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JP2008505635A Active JP5702519B2 (ja) 2005-04-07 2006-04-07 Aavベクターの機能を上げる方法
JP2012055665A Active JP5677681B2 (ja) 2005-04-07 2012-03-13 改変されたaavベクター
JP2014102248A Active JP5912147B2 (ja) 2005-04-07 2014-05-16 改変されたaavベクター
JP2014244953A Active JP6039632B2 (ja) 2005-04-07 2014-12-03 Aavベクターの機能を上げる方法
JP2016093699A Active JP6275769B2 (ja) 2005-04-07 2016-05-09 Aavベクターの機能を上げる方法

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JP2012055665A Active JP5677681B2 (ja) 2005-04-07 2012-03-13 改変されたaavベクター
JP2014102248A Active JP5912147B2 (ja) 2005-04-07 2014-05-16 改変されたaavベクター
JP2014244953A Active JP6039632B2 (ja) 2005-04-07 2014-12-03 Aavベクターの機能を上げる方法
JP2016093699A Active JP6275769B2 (ja) 2005-04-07 2016-05-09 Aavベクターの機能を上げる方法

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US (15) US8999678B2 (enExample)
EP (12) EP1866422B1 (enExample)
JP (5) JP5702519B2 (enExample)
CN (6) CN117363655A (enExample)
DE (2) DE23193601T1 (enExample)
DK (2) DK2359867T3 (enExample)
ES (6) ES2525143T3 (enExample)
WO (1) WO2006110689A2 (enExample)

Families Citing this family (410)

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Publication number Priority date Publication date Assignee Title
NZ618298A (en) 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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
HUE035567T2 (en) 2003-09-30 2018-05-28 Univ Pennsylvania Adeno-associated virus (AAV) clusters, sequences, vectors and applications containing them
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
US8198421B2 (en) * 2006-06-19 2012-06-12 Asklepios Biopharmaceutical, Inc. Modified factor VIII and factor IX genes and vectors for gene therapy
HRP20161150T1 (hr) 2007-04-09 2016-11-18 University Of Florida Research Foundation, Inc. PRIPRAVCI rAAV VEKTORA KOJI IMAJU TIROZIN-MODIFICIRANE CAPSID PROTEINE I POSTUPCI ZA UPORABU
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
WO2010011404A2 (en) 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
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
US10920244B2 (en) 2009-04-30 2021-02-16 The Trustees Of The University Of Pennsylvania Compositions for targeting conducting airway cells comprising adeno-associated virus constructs
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2011038187A1 (en) 2009-09-25 2011-03-31 The Trustees Of The University Of Pennsylvania Controlled adeno-associated virus (aav) diversification and libraries prepared therefrom
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of 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
JP5963743B2 (ja) 2010-04-23 2016-08-03 ユニバーシティ オブ マサチューセッツ Cnsターゲティングaavベクターおよびその使用方法
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
CA2833912C (en) 2010-04-23 2021-09-21 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
JP5704361B2 (ja) * 2010-10-27 2015-04-22 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
EP2699688A1 (en) 2011-04-20 2014-02-26 The Trustees Of The University Of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
US9226976B2 (en) 2011-04-21 2016-01-05 University Of Massachusetts RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
MX349138B (es) 2011-04-22 2017-07-13 Univ California Variones de virus adeno-asociados con capside variante y sus metodos de uso.
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
RU2653444C2 (ru) * 2012-02-17 2018-05-08 Дзе Чилдрен'З Хоспитал Оф Филадельфия Композиции вектора aav и способы переноса генов в клетки, органы и ткани
HUE054087T2 (hu) * 2012-04-18 2021-09-28 Childrens Hospital Philadelphia Készítmények és eljárások nagy hatékonyságú géntranszferre AAV kapszidvariánsok alkalmazásával
TWI698240B (zh) 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
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
US20150252384A1 (en) 2012-08-01 2015-09-10 National 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
PL2900686T3 (pl) * 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
MX386059B (es) 2013-03-15 2025-03-18 Univ Pennsylvania COMPOSICIONES Y SUS usos EN EL TRATAMIENTO DE LA MUCOPOLISACARIDOSIS TIPO I.
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
CA2917018A1 (en) * 2013-05-21 2014-12-04 University Of Florida Research Foundation, Inc. Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer
CA2907799A1 (en) * 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
SI3024498T1 (sl) * 2013-07-22 2020-07-31 The Children's Hospital Of Philadelphia Različica AAV in sestavki, postopki in uporabe za prenos genov v celice, organe in tkiva
EP3049527A4 (en) 2013-09-26 2017-08-16 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
WO2015054258A1 (en) * 2013-10-07 2015-04-16 Kiromic, Llc Compositions and methods for treating cardiovascular diseases using disease-specific promoter
EP3459965B1 (en) * 2013-10-11 2020-12-02 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
MX377804B (es) 2014-03-09 2025-03-11 Univ Pennsylvania Composiciones utiles en el tratamiento de deficiencia de la ornitina transcarbamilasa (otc).
KR102288849B1 (ko) 2014-03-17 2021-08-12 애드베룸 바이오테크놀로지스, 인코포레이티드 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법
US10280418B2 (en) 2014-03-18 2019-05-07 Univeristy Of Massachusetts RAAV-based compositions and methods for treating amyotrophic lateral sclerosis
EP3134431B1 (en) 2014-04-25 2021-04-07 The Trustees Of The University Of Pennsylvania Ldlr variants and their use in compositions for reducing cholesterol levels
EP3134115A4 (en) 2014-04-25 2017-10-25 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
PT3137497T (pt) 2014-05-02 2021-07-12 Genzyme Corp Vetores de aav para terapia genética na retina e snc
HRP20201530T1 (hr) 2014-05-13 2020-12-11 The Trustees Of The University Of Pennsylvania Kompozicije koje sadrže aav koji eksprimira konstrukte dvojnih antitijela i njihove primjene
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
AU2015301598B2 (en) * 2014-08-13 2020-07-16 Liron Elkouby An improved expression cassette for packaging and expression of variant Factor VIII for the treatment of hemostasis disorders
US10973931B2 (en) 2014-09-16 2021-04-13 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
JP6842410B2 (ja) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるaavベクター
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
WO2016073693A2 (en) 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
CA2975583A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
CA2967393A1 (en) 2014-11-21 2016-05-26 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system
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
EP3978614A3 (en) 2015-01-07 2022-07-27 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
JP7095994B2 (ja) 2015-03-02 2022-07-05 アドヴェラム バイオテクノロジーズ, インコーポレイテッド ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法
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
CA2979065A1 (en) 2015-03-24 2016-09-29 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
CN107531760A (zh) * 2015-03-30 2018-01-02 勃林格殷格翰动物保健公司 Pcv2 orf2载体平台
US20180110877A1 (en) 2015-04-27 2018-04-26 The Trustees Of The University Of Pennsylvania DUAL AAV VECTOR SYSTEM FOR CRISPR/Cas9 MEDIATED CORRECTION OF HUMAN DISEASE
AU2016256894B2 (en) 2015-05-07 2020-11-26 Massachusetts Eye And Ear Infirmary Methods of delivering an agent to the eye
US11535665B2 (en) 2015-05-13 2022-12-27 The Trustees Of The University Of Pennsylvania AAV-mediated expression of anti-influenza antibodies and methods of use thereof
IL309741A (en) 2015-06-23 2024-02-01 Childrens Hospital Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
ES3009026T3 (en) 2015-07-22 2025-03-25 Univ Duke High-throughput screening of regulatory element function with epigenome editing technologies
JP7166168B2 (ja) * 2015-07-30 2022-11-07 マサチューセッツ アイ アンド イヤー インファーマリー 祖先ウイルス配列およびその使用
WO2017023801A1 (en) 2015-07-31 2017-02-09 Regents Of The University Of Minnesota Intracellular genomic transplant and methods of therapy
KR102745604B1 (ko) 2015-08-06 2024-12-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Glp-1 및 대사성 질환 치료용 조성물에서 이의 용도
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
KR20180057636A (ko) * 2015-08-31 2018-05-30 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 반려 동물 치료용 aav-epo
JP7261583B2 (ja) 2015-09-24 2023-04-20 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 補体媒介性疾患を処置するための組成物及び方法
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
EP3364996B1 (en) 2015-10-22 2021-08-25 University of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
DK3364997T5 (da) 2015-10-22 2024-09-30 Univ Massachusetts Aspartoacylase genterapi til behandling af canavans sygdom
US12188037B2 (en) * 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
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
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
CA3006569A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
IL259850B2 (en) 2015-12-11 2023-11-01 Univ Pennsylvania Gene therapy for treating familial hypercholesterolemia
EP3387118B1 (en) 2015-12-11 2022-04-06 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
EP3387117B1 (en) 2015-12-11 2022-11-23 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
CA3007476A1 (en) 2015-12-11 2017-06-15 Massachusetts Eye And Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
WO2017106345A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Composition for treatment of crigler-najjar syndrome
ES2826384T3 (es) 2015-12-14 2021-05-18 Univ Pennsylvania Terapia génica para trastornos oculares
CA3008013A1 (en) 2015-12-14 2017-06-22 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
EP3411059A4 (en) * 2016-02-02 2019-10-16 University Of Massachusetts METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM
IL260959B2 (en) 2016-02-03 2024-09-01 Univ Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
EP3443001B1 (en) 2016-04-11 2025-04-30 Obsidian Therapeutics, Inc. REGULATED BIOCIRCUIT SYSTEMS
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
US11197937B2 (en) 2016-04-15 2021-12-14 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
SG11201808176TA (en) 2016-04-15 2018-10-30 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
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
US11401527B2 (en) 2016-04-17 2022-08-02 The Trustees Of The University Of Pennsylvania Compositions and methods useful for prophylaxis of organophosphates
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
RU2725286C2 (ru) 2016-05-13 2020-06-30 4Д Молекьюлар Терапьютикс Инк. Варианты капсидов аденоассоциированного вируса и способы их применения
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
IL262784B2 (en) 2016-05-18 2023-10-01 Voyager Therapeutics Inc modulatory polynucleotides
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
WO2018009814A1 (en) 2016-07-08 2018-01-11 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving rdh12
US12214056B2 (en) 2016-07-19 2025-02-04 Duke University Therapeutic applications of CPF1-based genome editing
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
EP3491008A2 (en) * 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
CN116286986A (zh) 2016-07-29 2023-06-23 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体和其使用方法
EP3500667A4 (en) 2016-08-18 2020-09-02 The Regents of the University of California GENOMIC ENGINEERING OF CRISPR-CAS THROUGH A MODULAR AAV DELIVERY SYSTEM
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
US11078238B2 (en) * 2016-09-29 2021-08-03 University Of Florida Research Foundation, Incorporated AAVrh.10 variants with host antibody escape capabilities and altered tissue targeting properties
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
EP4338799A3 (en) 2016-10-18 2024-06-05 Regents of the University of Minnesota Tumor infiltrating lymphocytes and methods of therapy
EP3528785A4 (en) 2016-10-19 2020-12-02 Adverum Biotechnologies, Inc. MODIFIED AAV CASPIDS AND USES THEREOF
US20190328846A1 (en) 2016-12-01 2019-10-31 INSERM (Institut National de la Santé et de la Recherche Médicale) Pharmaceutical compositions for the treatment of retinal degenerative diseases
WO2018139634A1 (ja) * 2017-01-30 2018-08-02 学校法人日本医科大学 アデノ随伴ウイルス(aav)キャプシドタンパク質の変異体
AU2018220212A1 (en) 2017-02-20 2019-08-08 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
CA3054131C (en) 2017-02-21 2021-05-25 University Of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
PT3589730T (pt) 2017-02-28 2024-02-28 Univ Pennsylvania Vetor de vírus adenoassociado (aav) de clado f e suas utilizações
CN110573523A (zh) 2017-02-28 2019-12-13 宾夕法尼亚州大学信托人 基于aav载体的流感疫苗
KR20240104196A (ko) 2017-03-01 2024-07-04 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 안구 장애에 대한 유전자 치료
KR20240093919A (ko) 2017-04-14 2024-06-24 리젠엑스바이오 인크. 인간 뉴런 또는 신경교 세포에 의해 생성된 재조합 인간 이두로네이트-2 설파타제(ids)를 이용한 뮤코다당증 ii형의 치료
EP3634986A4 (en) 2017-04-24 2021-09-08 The Trustees of The University of Pennsylvania GENE THERAPY FOR EYE DISORDERS
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
AU2018265414B2 (en) 2017-05-10 2022-01-20 Massachusetts Eye And Ear Infirmary Methods and compositions for modifying assembly-activating protein (APP)-dependence of viruses
CN108103082B (zh) * 2017-05-11 2021-07-13 北京锦篮基因科技有限公司 一种腺相关病毒介导的lcat基因表达载体及其用途
SG11201910015SA (en) 2017-05-11 2019-11-28 Univ Pennsylvania Gene therapy for neuronal ceroid lipofuscinoses
WO2018218359A1 (en) 2017-05-31 2018-12-06 The Trustees Of The University Of Pennsylvania Gene therapy for treating peroxisomal disorders
WO2018232149A1 (en) 2017-06-14 2018-12-20 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
AU2018290954B2 (en) 2017-06-30 2023-07-13 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
EP3645021A4 (en) * 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
BR112020000063A2 (pt) 2017-07-06 2020-07-14 The Trustees Of The University Of Pennsylvania terapia gênica mediada por aav9 para tratamento de mucopolissacaridose tipo i
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
AU2018311069A1 (en) 2017-08-03 2020-02-20 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
AU2018320849A1 (en) * 2017-08-25 2020-03-05 Ovid Therapeutics Inc. Recombinant adeno-associated vectors
EP3676373A4 (en) 2017-08-28 2021-06-09 The Regents of The University of California Adeno-associated virus capsid variants and methods of use thereof
KR20250011726A (ko) * 2017-09-20 2025-01-21 4디 몰레큘러 테라퓨틱스 아이엔씨. 아데노-관련 바이러스 변이체 캡시드 및 그 용도
AU2018335752B2 (en) 2017-09-22 2025-04-10 Christian HINDERER Gene therapy for treating Mucopolysaccharidosis type ii
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
CA3079568A1 (en) 2017-10-18 2019-04-25 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
EP3697449A1 (en) 2017-10-18 2020-08-26 REGENXBIO Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified vegf-trap
CR20200282A (es) 2017-11-27 2021-02-18 4D Molecular Therapeutics Inc Cápsides variantes de virus adenoasociados y su uso para inhibir la angiogénesis
JP7384797B2 (ja) 2017-11-30 2023-11-21 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iiib型のための遺伝子療法
WO2019108857A1 (en) 2017-11-30 2019-06-06 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis iiia
BR112020012336A2 (pt) 2017-12-19 2021-03-30 Akouos, Inc. Administração de anticorpos terapêuticos mediada por aav para o ouvido interno
US10806802B2 (en) 2018-01-18 2020-10-20 University Of Guelph Adeno-associated virus particle with mutated capsid and methods of use thereof
US12305185B2 (en) 2018-01-23 2025-05-20 Mount Spec Investments Pty Ltd Expression vector and method
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
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
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
CN112352050A (zh) * 2018-02-27 2021-02-09 宾夕法尼亚州大学信托人 新颖腺相关病毒(aav)载体、具有降低的衣壳脱酰胺化的aav载体及其用途
KR102848518B1 (ko) * 2018-02-27 2025-08-22 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 캡시드 탈아미드화가 감소된 신규 아데노-연관 바이러스(aav) 벡터 및 이의 용도
WO2019191418A1 (en) * 2018-03-29 2019-10-03 Asklepios Biopharmaceutical, Inc. Liver tropic recombinant aav6 vectors that evade neutralization
BR112020020223A2 (pt) 2018-04-05 2021-01-19 Genethon Sorotipo de vírus adeno-associado híbrido recombinante entre aav9 e aavrh74 com tropismo pelo fígado reduzido
WO2019195701A1 (en) 2018-04-05 2019-10-10 Massachusetts Eye And Ear Infirmary Methods of making and using combinatorial barcoded nucleic acid libraries having defined variation
CN112601454B (zh) 2018-04-16 2023-10-27 宾夕法尼亚州大学信托人 用于治疗杜兴肌营养不良的组合物和方法
KR20210009317A (ko) 2018-04-18 2021-01-26 더 트러스티스 오브 콜롬비아 유니버시티 인 더 시티 오브 뉴욕 미토콘드리아 dna 고갈 증후군을 포함하는 불균형 뉴클레오타이드 풀에 의해 초래된 질환에 대한 유전자 요법
CA3098565A1 (en) 2018-04-29 2019-11-07 Claire G. ZHANG Scalable clarification process for recombinant aav production
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
US20210207168A1 (en) 2018-05-08 2021-07-08 Rutgers, The State University Of New Jersey Aav-compatible laminin-linker polymerization proteins
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
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US20220403001A1 (en) 2018-06-12 2022-12-22 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
EP3807404A1 (en) 2018-06-13 2021-04-21 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
IL319002A (en) 2018-06-14 2025-04-01 Regenxbio Inc Anion exchange chromatography for recombinant AAV production
GB201811368D0 (en) 2018-07-11 2018-08-29 Ucb Biopharma Sprl Antibody
JP7413629B2 (ja) 2018-07-17 2024-01-16 ヘリックスミス カンパニー, リミテッド Igf-1異型体を発現させるdnaコンストラクトを用いた神経病症の治療
JP2021530548A (ja) 2018-07-24 2021-11-11 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics, Inc. 遺伝子治療製剤を生産するための系および方法
US20210301306A1 (en) * 2018-07-31 2021-09-30 Cornell University Gene therapy methods to control organ function
WO2020033842A1 (en) 2018-08-10 2020-02-13 Regenxbio Inc. Scalable method for recombinant aav production
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US12195745B2 (en) * 2018-08-21 2025-01-14 Massachusetts Eye And Ear Infirmary Compositions and methods for modulating transduction efficiency of adeno-associated viruses
BR112021003897A2 (pt) 2018-08-30 2021-05-25 Tenaya Therapeutics, Inc. reprogramação de células cardíacas com miocarina e asci1
SG11202103151RA (en) 2018-09-28 2021-04-29 Voyager Therapeutics Inc Frataxin expression constructs having engineered promoters and methods of use thereof
MX2021003869A (es) 2018-10-01 2021-09-08 Univ Pennsylvania Composiciones útiles para tratar la gangliosidosis gm1.
US12472268B2 (en) 2018-10-01 2025-11-18 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
TW202035689A (zh) 2018-10-04 2020-10-01 美商航海家醫療公司 測量病毒載體粒子的效價及強度之方法
CN113166731A (zh) 2018-10-05 2021-07-23 沃雅戈治疗公司 编码aav生产蛋白的工程化核酸构建体
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
AU2019362833A1 (en) 2018-10-15 2021-04-29 Regenxbio Inc. Method for measuring the infectivity of replication defective viral vectors and viruses
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
EP3867389A1 (en) 2018-10-15 2021-08-25 Voyager Therapeutics, Inc. Expression vectors for large-scale production of raav in the baculovirus/sf9 system
EP3870600A1 (en) 2018-10-24 2021-09-01 Obsidian Therapeutics, Inc. Er tunable protein regulation
EP3890786A4 (en) 2018-12-05 2022-08-31 Abeona Therapeutics Inc. RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR FOR GENE RELEASE
PT3906066T (pt) 2019-01-04 2024-02-06 Ultragenyx Pharmaceutical Inc Constructos de terapia génica para o tratamento da doença de wilson
CA3125770A1 (en) 2019-01-18 2020-07-23 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
WO2020154535A1 (en) * 2019-01-23 2020-07-30 University Of Florida Research Foundation, Incorporated Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design
EP3917566A4 (en) 2019-01-31 2022-10-26 Oregon Health & Science University METHODS OF USING TRANSCRIPTION-DEPENDENT DIRECTED EVOLUTION OF AAV CAPSIDS
JP7637058B2 (ja) 2019-02-22 2025-02-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Grn関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス
JP2022521025A (ja) 2019-02-25 2022-04-04 ノバルティス アーゲー Biettiクリスタリン網膜症を治療するための組成物及び方法
US20220154211A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
CN114040980A (zh) 2019-02-26 2022-02-11 宾夕法尼亚州大学信托人 可用于治疗克拉伯病的组合物
AU2020253435A1 (en) 2019-04-01 2021-11-18 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
AU2020253462A1 (en) 2019-04-03 2021-10-28 Regenxbio Inc. Gene therapy for eye pathologies
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
SI3953483T1 (sl) 2019-04-11 2024-02-29 Regenxbio Inc. Postopki gelske izključitvene kromatografije za karakterizacijo spojin rekombinantnih adeno-povezanih virusov
AR118734A1 (es) 2019-04-19 2021-10-27 Regenxbio Inc Formulaciones y métodos de vectores de virus adenoasociados
EP3959325A2 (en) * 2019-04-23 2022-03-02 INSERM (Institut National de la Santé et de la Recherche Médicale) New adeno-associated virus (aav) variants and uses thereof for gene therapy
SG11202111414RA (en) 2019-04-24 2021-11-29 Regenxbio Inc Fully-human post-translationally modified antibody therapeutics
EP3752524A4 (en) * 2019-04-27 2021-11-24 Ocugen, Inc. GENE THERAPY MEDIATED BY AN ADENO-ASSOCIATED VIRUS VECTOR FOR OPHTHALMIC DISEASES
JP7644026B2 (ja) * 2019-04-29 2025-03-11 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 新規aavカプシドおよびそれを含む組成物
KR20220020261A (ko) 2019-05-03 2022-02-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 이염성 백질이영양증의 치료에 유용한 조성물
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
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
US11319354B2 (en) 2019-07-10 2022-05-03 Masonic Medical Research Laboratory VGLL4 with UCP-1 cis-regulatory element and method of use thereof
WO2021007515A1 (en) 2019-07-11 2021-01-14 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with micrornas and other factors
RS66255B1 (sr) * 2019-07-15 2024-12-31 Meiragtx Uk Ii Ltd Modifikovani proteini aav kapsida za lečenje artritične bolesti
EP4007814A1 (en) 2019-07-26 2022-06-08 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
CA3145112A1 (en) 2019-07-26 2021-02-04 Chunping Qiao Engineered nucleic acid regulatory element and methods of uses thereof
MX2022002366A (es) 2019-08-26 2022-07-19 Regenxbio Inc Tratamiento de la retinopatía diabética con fab anti-vegf completamente humano modificado postraduccionalmente.
US20230092895A1 (en) 2019-08-30 2023-03-23 Obsidian Therapeutics, Inc. Tandem cd19 car-based compositions and methods for immunotherapy
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
WO2021046451A1 (en) 2019-09-06 2021-03-11 Obsidian Therapeutics, Inc. Compositions and methods for dhfr tunable protein regulation
CA3150330A1 (en) 2019-09-13 2021-03-18 Peter D. YURCHENCO Aav-compatible laminin-linker polymerization proteins
US20220347298A1 (en) 2019-10-04 2022-11-03 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
AU2020362119A1 (en) 2019-10-07 2022-05-26 Regenxbio Inc. Adeno-associated virus vector pharmaceutical composition and methods
EP4045664A1 (en) * 2019-10-16 2022-08-24 Wuxi Apptec (Shanghai) Co., Ltd. A novel aav variant
US20230016983A1 (en) 2019-11-19 2023-01-19 lNSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) Antisense oligonucleotides and thier use for the treatment of cancer
BR112022009895A2 (pt) 2019-11-28 2022-08-09 Regenxbio Inc Construtos de terapia de gene de microdistrofina e usos dos mesmos
CA3161367A1 (en) * 2019-12-10 2021-06-17 Homology Medicines, Inc. Adeno-associated virus compositions and methods of use thereof
IL293684A (en) 2019-12-10 2022-08-01 Takeda Pharmaceuticals Co Adeno associated virus vectors for the treatment of hunter disease
JP2023509443A (ja) 2019-12-31 2023-03-08 スワンバイオ セラピューティクス リミテッド 改善されたaav-abcd1コンストラクトならびに副腎白質ジストロフィー(ald)および/または副腎脊髄ニューロパチー(amn)の処置または予防のための使用
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
CA3167685A1 (en) 2020-01-22 2021-07-29 Regenxbio Inc. Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
JP2023512242A (ja) 2020-01-29 2023-03-24 レジェンクスバイオ インコーポレーテッド ヒト神経細胞またはヒトグリア細胞が産生した組換えヒトイズロン酸-2-スルファターゼ(ids)によるムコ多糖症iiの治療
AU2021212749A1 (en) 2020-01-29 2022-08-18 Regenxbio Inc. Treatment of mucopolysaccharidosis IVA
MX2022009462A (es) 2020-02-02 2022-11-10 Univ Pennsylvania Composiciones útiles para tratar la gangliosidosis gm1.
BR112022015921A2 (pt) 2020-02-14 2022-10-04 Ultragenyx Pharmaceutical Inc Terapia gênica para tratar o transtorno de deficiência de cdkl5
TW202142554A (zh) * 2020-02-25 2021-11-16 澳洲兒童醫學研究所 腺相關病毒蛋白殼多肽及載體
US20230235353A1 (en) 2020-03-19 2023-07-27 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
WO2021191424A1 (en) 2020-03-27 2021-09-30 UCB Biopharma SRL Autonomous knob domain peptides
EP4127189A1 (en) 2020-03-31 2023-02-08 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
WO2021211629A2 (en) * 2020-04-13 2021-10-21 Massachusetts Eye And Ear Infirmary Methods of making and using a vaccine against coronavirus
KR20230020394A (ko) 2020-04-15 2023-02-10 보이저 테라퓨틱스, 인크. Tau 결합 화합물
JP2023526310A (ja) 2020-05-12 2023-06-21 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 導入遺伝子発現のdrg特異的低減のための組成物
AU2021271635A1 (en) 2020-05-12 2023-01-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
KR20230041965A (ko) 2020-05-13 2023-03-27 아카우오스, 인크. Slc26a4-연관 청력손실을 치료하기 위한 조성물 및 방법
EP4165194A2 (en) 2020-05-13 2023-04-19 Akouos, Inc. Compositions and methods for treating kcnq4-associated hearing loss
CA3173450A1 (en) 2020-05-26 2021-12-02 Francois Vigneault High throughput engineering of functional aav capsids
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
CN115968302A (zh) 2020-06-17 2023-04-14 宾夕法尼亚州大学信托人 用于治疗基因疗法患者的组合物和方法
JP2023532704A (ja) * 2020-07-03 2023-07-31 ジェネトン 超可変領域スワッピングを通じて新規のハイブリッドaavカプシドを操作する方法
WO2022011262A1 (en) 2020-07-10 2022-01-13 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods and compositions for treating epilepsy
MX2023000658A (es) 2020-07-13 2023-02-23 Univ Pennsylvania Composiciones utiles para el tratamiento de la enfermedad de charcot-marie-tooth.
AU2021315876A1 (en) 2020-07-27 2023-02-23 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
CN116121274B (zh) * 2020-07-29 2024-11-19 北京三诺佳邑生物技术有限责任公司 一组肝靶向新型腺相关病毒的获得及其应用
AU2021319208A1 (en) 2020-07-30 2023-03-02 Shape Therapeutics Inc. Stable cell lines for inducible production of rAAV virions
WO2022046815A1 (en) 2020-08-24 2022-03-03 The Trustees Of The University Of Pennsylvania Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
WO2022046988A1 (en) 2020-08-26 2022-03-03 The Trustees Of The University Of Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
EP4213890A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized lanadelumab and administration thereof
WO2022060916A1 (en) 2020-09-15 2022-03-24 Regenxbio Inc. Vectorized antibodies for anti-viral therapy
WO2022061002A1 (en) 2020-09-18 2022-03-24 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
US20230364206A1 (en) 2020-10-07 2023-11-16 Regenxbio Inc. Gene therapy for ocular manifestations of cln2 disease
AU2021358048A1 (en) 2020-10-07 2023-05-25 Regenxbio Inc. Formulations for suprachoroidal administration such as high viscosity formulations
WO2022076591A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
US20230414788A1 (en) 2020-10-07 2023-12-28 Regenxbio Inc. Formulations for suprachoroidal administration such as gel formulations
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
JP2023545722A (ja) 2020-10-07 2023-10-31 リジェネックスバイオ インコーポレイテッド 遺伝子治療剤の眼送達のためのアデノ随伴ウイルス
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
KR20230118075A (ko) 2020-10-09 2023-08-10 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 파브리병 치료를 위한 조성물 및 방법
CA3195553A1 (en) 2020-10-18 2022-04-21 Qiang Wang Improved adeno-associated virus (aav) vector and uses therefor
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
EP4236999A1 (en) 2020-10-28 2023-09-06 RegenxBio Inc. Vectorized anti-tnf-alfa antibodies for ocular indications
KR20230098176A (ko) 2020-10-29 2023-07-03 리젠엑스바이오 인크. 안구 적응증을 위한 벡터화된 tnf-알파 길항제
US20230390418A1 (en) 2020-10-29 2023-12-07 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
TW202237850A (zh) 2020-12-01 2022-10-01 賓州大學委員會 具有組織特異性靶向基序的新穎構成物及含有其之組成物
MX2023006445A (es) 2020-12-01 2023-08-10 Univ Pennsylvania Composiciones y usos de estas para el tratamiento del síndrome de angelman.
AU2021391433A1 (en) 2020-12-01 2023-06-22 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
AU2021403076A1 (en) 2020-12-16 2023-06-29 Regenxbio Inc. Method of producing a recombinant adeno-associated virus particle
CA3203373A1 (en) 2020-12-29 2022-07-07 Emmanuel John Simons Compositions and methods for treating clrn1-associated hearing loss and/or vision loss
US20240309076A1 (en) 2020-12-29 2024-09-19 Regenxbio Inc. Tau-specific antibody gene therapy compositions, methods and uses thereof
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
AU2022214429A1 (en) 2021-02-01 2023-09-14 Tern Therapeutics, Llc 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
US20240117328A1 (en) 2021-02-10 2024-04-11 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
CA3209673A1 (en) 2021-02-26 2022-09-01 Rizwana ISLAM Composition and methods for the treatment of fabry disease
CA3215141A1 (en) 2021-04-12 2022-10-20 James M. Wilson Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
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
MX2023012511A (es) 2021-04-23 2024-01-08 Univ Rochester Edición del genóma por inserción no homóloga de adn dirigida utilizando una proteína de fusión retroviral de integrasa-cas y métodos de tratamiento.
TW202309293A (zh) 2021-04-26 2023-03-01 美商銳進科斯生物股份有限公司 用於治療肌縮蛋白症(dystrophinopathy)之微小肌縮蛋白基因療法給藥
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
EP4359549A1 (en) 2021-06-25 2024-05-01 Oxford Biomedica (US) LLC Adeno-associated virus packaging systems
AU2022301302A1 (en) 2021-07-01 2024-01-25 Indapta Therapeutics, Inc. Engineered natural killer (nk) cells and related methods
US20240409955A1 (en) 2021-07-19 2024-12-12 New York University Auf1 combination therapies for treatment of muscle degenerative disease
MX2024001208A (es) 2021-08-11 2024-04-22 Sana Biotechnology Inc Celulas primarias geneticamente modificadas para terapia celular alogenica.
EP4384544A1 (en) 2021-08-11 2024-06-19 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy
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
JP2024531138A (ja) 2021-08-11 2024-08-29 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 筋ジストロフィーを処置するための組成物および方法
AU2022325231A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions
WO2023028513A1 (en) 2021-08-24 2023-03-02 Oxford Biomedica Solutions Llc Adeno-associated virus formulations
AU2022335593A1 (en) * 2021-08-25 2024-03-14 Canbridge Pharmaceuticals, Inc. Aav particles comprising liver-tropic capsid protein and acid alpha-glucosidase and use to treat pompe disease
IL311096A (en) 2021-08-31 2024-04-01 Scout Bio Inc Antigen binding molecules and their uses
IL311572A (en) 2021-09-30 2024-05-01 Akouos Inc Compositions and methods for treating kcnq4-associated hearing loss
US20240384298A1 (en) 2021-10-02 2024-11-21 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
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
WO2023060269A1 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
WO2023060272A2 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
WO2023072181A1 (en) * 2021-10-28 2023-05-04 Shanghai Vitalgen Biopharma Co., Ltd. Adeno-associated virus capsid
WO2023077092A1 (en) 2021-10-28 2023-05-04 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
US20230183658A1 (en) 2021-10-29 2023-06-15 Oxford Biomedica Solutions Llc Methods and compositions for the purification of adeno-associated virus
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
JP2024543918A (ja) 2021-11-29 2024-11-26 上海瑞宏迪医薬有限公司 Aadc、gdnfポリヌクレオチド及びそのパーキンソン病を治療するための使用
CA3239452A1 (en) 2021-12-01 2023-06-08 Thomas Packard Functional aav capsids for systemic administration
US20250026795A1 (en) 2021-12-01 2025-01-23 Shape Therapeutics Inc. Functional aav capsids for intravitreal administration
US20240408157A1 (en) 2021-12-02 2024-12-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
CN118510905A (zh) * 2021-12-06 2024-08-16 学校法人顺天堂 改变型腺伴随病毒载体
WO2023114897A2 (en) 2021-12-15 2023-06-22 Homology Medicines, Inc. Methods and compositions for the production of adeno-associated virus
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
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
TW202338086A (zh) 2022-01-10 2023-10-01 賓州大學委員會 有用於治療異染性白質失養症之組成物
WO2023147304A1 (en) 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
EP4473011A1 (en) 2022-02-02 2024-12-11 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
JP2025508720A (ja) 2022-02-17 2025-04-10 サナ バイオテクノロジー,インコーポレイテッド 遺伝子操作されたcd47タンパク質及びその使用関連出願の相互参照
CA3244192A1 (en) 2022-02-21 2025-01-22 Shanghai Regenelead Therapies Co Ltd VEGF-BINDING MOLECULE AND ITS PHARMACEUTICAL USE
TW202342740A (zh) 2022-03-07 2023-11-01 美商奧崔基尼克斯製藥公司 改良的批量aav生產系統和方法
US20250101382A1 (en) 2022-03-11 2025-03-27 Sana Biotechnology, Inc. Genetically modified cells and compositions and uses thereof
TW202346590A (zh) 2022-03-13 2023-12-01 美商銳進科斯生物股份有限公司 經修飾之肌肉特異性啟動子
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
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
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
US20250250326A1 (en) 2022-04-06 2025-08-07 The Trustees Of The University Of Pennsylvania Passive immunization with anti-aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
CN119317645A (zh) 2022-04-06 2025-01-14 宾夕法尼亚州大学信托人 用于治疗her2阳性转移性乳腺癌及其他癌症的组合物和方法
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
WO2023201308A1 (en) 2022-04-14 2023-10-19 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
EP4518972A2 (en) 2022-05-03 2025-03-12 RegenxBio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
TW202417633A (zh) 2022-05-03 2024-05-01 美商銳進科斯生物股份有限公司 用於眼適應症之載體化抗TNF-α抑制劑
CA3255627A1 (en) 2022-05-06 2023-11-09 Novartis Ag NEW VP2 FUSION POLYPEPTIDES OF RECOMBINANT AAV
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
CN119731321A (zh) 2022-06-24 2025-03-28 图恩疗法股份有限公司 通过靶向基因阻遏减少低密度脂蛋白的组合物、系统和方法
JP2025525439A (ja) 2022-06-30 2025-08-05 インダプタ セラピューティクス インコーポレイテッド 操作されたナチュラルキラー(nk)細胞と抗体療法との組み合わせおよび関連する方法
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
WO2024044725A2 (en) 2022-08-24 2024-02-29 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
WO2024042485A1 (en) 2022-08-25 2024-02-29 Takeda Pharmaceutical Company Limited Composition for use in the treatment of fabry disease
WO2024073669A1 (en) 2022-09-30 2024-04-04 Regenxbio Inc. Treatment of ocular diseases with recombinant viral vectors encoding anti-vegf fab
CN115960177B (zh) * 2022-10-09 2023-07-07 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2024081746A2 (en) 2022-10-11 2024-04-18 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2024105638A1 (en) 2022-11-18 2024-05-23 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of hunter syndrome
CN115838399B (zh) * 2022-12-08 2023-08-11 广州派真生物技术有限公司 腺相关病毒突变体及其应用
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
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
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
WO2024163012A1 (en) 2023-02-02 2024-08-08 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024192281A2 (en) 2023-03-15 2024-09-19 Regenxbio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
WO2024211780A1 (en) 2023-04-07 2024-10-10 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
AU2024264889A1 (en) 2023-05-03 2025-11-13 Sana Biotechnology, Inc. Methods of dosing and administration of engineered islet cells
WO2024229309A2 (en) 2023-05-03 2024-11-07 Manifold Biotechnologies, Inc. Methodsand compositions for high-throughput protein delivery, screening, and detection
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
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof
WO2024238853A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
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
AR133098A1 (es) 2023-06-29 2025-08-27 Univ Pennsylvania Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen
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
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
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
WO2025082618A1 (en) 2023-10-20 2025-04-24 Fuse Vectors Aps In vitro parvovirus capsid manufacturing
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
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
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
WO2025188625A1 (en) 2024-03-03 2025-09-12 Passage Bio, Inc. Recombinant adeno-associated virus for treatment of neurodegenerative disorders
US20250276095A1 (en) 2024-03-04 2025-09-04 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
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
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
CN118460614B (zh) * 2024-07-05 2025-03-11 凌意(杭州)生物科技有限公司 一种腺相关病毒Cap蛋白的表达框
CN118754952B (zh) * 2024-09-06 2025-01-10 上海玮美基因科技有限责任公司 一种新型腺相关病毒及其应用

Family Cites Families (71)

* 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
IL74909A (en) 1984-04-20 1992-01-15 Genentech Inc Preparation of functional human factor viii and dna sequences,expression vectors,transformed microorganisms and cell lines used therein
US4965199A (en) 1984-04-20 1990-10-23 Genentech, Inc. Preparation of functional human factor VIII in mammalian cells using methotrexate based selection
EP0182448A3 (en) 1984-08-24 1987-10-28 Genetics Institute, Inc. Production of factor viii and related products
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
FI98829C (fi) 1986-01-27 1997-08-25 Chiron Corp Menetelmä rekombinoidun proteiinikompleksin valmistamiseksi, jolla on humaanitekijä VIII:C-aktiivisuutta
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
US5171844A (en) 1987-06-12 1992-12-15 Gist-Brocades N.W. 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
EP0500734B1 (en) 1989-11-17 1998-02-11 Novo Nordisk A/S Protein complexes having factor viii:c activity and production thereof
SE465222C5 (sv) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning
SE468050C (sv) 1991-03-15 1998-04-27 Pharmacia & Upjohn Ab Rekombinant derivat av human faktor VIII
US5661008A (en) 1991-03-15 1997-08-26 Kabi Pharmacia Ab Recombinant human factor VIII derivatives
CA2078721A1 (en) 1991-09-24 1993-03-25 Hiroshi Yonemura Process for preparing human coagulation factor viii protein complex
US6174666B1 (en) 1992-03-27 2001-01-16 The United States Of America As Represented By The Department Of Health And Human Services Method of eliminating inhibitory/instability regions from mRNA
US5563045A (en) 1992-11-13 1996-10-08 Genetics Institute, Inc. Chimeric procoagulant proteins
WO1994011013A1 (en) 1992-11-13 1994-05-26 Duke University Chimeric blood coagulation 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
AU6486196A (en) 1995-07-11 1997-02-10 Chiron Corporation Novel factor viii:c polypeptide analogs with altered protease sites
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The 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
AU9397098A (en) 1997-09-19 1999-04-12 Trustees Of The University Of Pennsylvania, The Methods and cell line useful for production of recombinant adeno-associated viruses
AU1449499A (en) 1997-10-31 1999-05-24 Maxygen, Incorporated Modification of virus tropism and host range by viral genome shuffling
US6410300B1 (en) 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
AU762220B2 (en) 1998-05-28 2003-06-19 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The AAV5 vector and uses thereof
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
ES2288037T3 (es) 1998-11-05 2007-12-16 The Trustees Of The University Of Pennsylvania Secuencia de acido nucleico del serotipo 1 de adenovirus asociado, vectores y celulas huesped que las contienen.
CA2348382C (en) 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Chimeric parvovirus vectors and methods of making and administering the same
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
US7314912B1 (en) 1999-06-21 2008-01-01 Medigene Aktiengesellschaft AAv scleroprotein, production and use thereof
CN1357050A (zh) * 1999-07-02 2002-07-03 昂尼克斯药物公司 用于治疗疾病的腺病毒载体
DE19933288A1 (de) 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
AU4565401A (en) * 2000-03-14 2001-09-24 Thomas Jefferson University Production of chimeric capsid vectors
AU5301201A (en) 2000-03-31 2001-10-15 Gen Hospital Corp Methods of modulating hair growth
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
EP1419245A4 (en) * 2001-07-13 2006-04-05 Univ Iowa Res Found ADENO-ASSOCIATED PSEUDOTYPE VIRUSES AND USES THEREOF
US6410330B1 (en) 2001-07-27 2002-06-25 Coulter International Corp. Method for measurement of nucleated red blood cells
US7647184B2 (en) 2001-08-27 2010-01-12 Hanall Pharmaceuticals, Co. Ltd High throughput directed evolution by rational mutagenesis
CA2467959C (en) * 2001-11-09 2009-03-10 Robert M. Kotin Production of adeno-associated virus in insect cells
NZ618298A (en) * 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2280694T3 (es) * 2002-04-29 2007-09-16 The Trustees Of The University Of Pennsylvania Metodo para la extraccion directa y la amplificacion de virus integrados del adn celular de tejidos.
WO2004020600A2 (en) 2002-08-28 2004-03-11 University Of Florida Modified aav
CA2499824C (en) 2002-09-30 2009-12-08 Canon Kabushiki Kaisha Liquid supply system, fluid communicating structure, ink supply system, and inkjet recording head utilizing the fluid communicating structure
JP2004157072A (ja) 2002-11-08 2004-06-03 Japan Science & Technology Agency 免疫反応測定に用いられる高感度磁性マーカー
EP1486567A1 (en) * 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
EP2657248B1 (en) * 2003-06-19 2017-03-22 Genzyme Corporation AAV virions with decreased immunoreactivity and uses therefor
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
HUE035567T2 (en) * 2003-09-30 2018-05-28 Univ Pennsylvania Adeno-associated virus (AAV) clusters, sequences, vectors and applications containing them
NZ555830A (en) * 2004-12-15 2009-01-31 Univ North Carolina Chimeric vectors
US8283151B2 (en) * 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
EP2016174A2 (en) 2006-04-28 2009-01-21 The Trustees of the University of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
US20120322861A1 (en) * 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
WO2013078316A1 (en) * 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
HUE054087T2 (hu) * 2012-04-18 2021-09-28 Childrens Hospital Philadelphia Készítmények és eljárások nagy hatékonyságú géntranszferre AAV kapszidvariánsok alkalmazásával
SI3024498T1 (sl) * 2013-07-22 2020-07-31 The Children's Hospital Of Philadelphia Različica AAV in sestavki, postopki in uporabe za prenos genov v celice, organe in tkiva
EP3459965B1 (en) * 2013-10-11 2020-12-02 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
IL309741A (en) 2015-06-23 2024-02-01 Childrens Hospital Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
JP7166168B2 (ja) * 2015-07-30 2022-11-07 マサチューセッツ アイ アンド イヤー インファーマリー 祖先ウイルス配列およびその使用
IL298001A (en) * 2020-05-13 2023-01-01 Voyager Therapeutics Inc Redirecting tropism of adeno-associated virus capsids

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