JP2015096070A5 - - Google Patents

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JP2015096070A5
JP2015096070A5 JP2014244953A JP2014244953A JP2015096070A5 JP 2015096070 A5 JP2015096070 A5 JP 2015096070A5 JP 2014244953 A JP2014244953 A JP 2014244953A JP 2014244953 A JP2014244953 A JP 2014244953A JP 2015096070 A5 JP2015096070 A5 JP 2015096070A5
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Japan
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aav vector
vector according
gene product
aav
aavrh32
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JP2014244953A
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JP2015096070A (ja
JP6039632B2 (ja
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Claims (11)

  1. 配列番号2のアミノ酸配列を有するAAVrh32/33キャプシドを含んでなるアデノ随伴ウイルス(AAV)ベクターであって、宿主細胞中で遺伝子産物の発現を制御する調節配列の制御下に該遺伝子産物をコードする導入遺伝子を保有する、AAVベクター。
  2. 遺伝子産物が免疫原または抗原である、請求項1に記載のAAVベクター。
  3. 遺伝子産物がウイルス抗原または免疫原である、請求項1または2に記載のAAVベクター。
  4. 調節配列が3’および5’逆方向末端反復配列を含んでなる、請求項1〜3のいずれか1項に記載のAAVベクター。
  5. 逆方向末端反復配列がAAVrh32/33に対して異種AAVからのものである、請求項4に記載のAAVベクター。
  6. 遺伝子産物が免疫グロブリンである、請求項1〜5のいずれか1項に記載のAAVベクター。
  7. 導入遺伝子ががん遺伝子ポリペプチドまたはがん遺伝子産物をコードする、AAVベクター。
  8. 製薬学的に許容され得る担体と請求項1〜7のいずれか1項に記載のAAVベクターを含んでなる組成物。
  9. 請求項1〜7に記載のAAVベクターによりインビトロでトランスフェクトされた宿主細胞。
  10. 配列番号2のアミノ酸配列を有するAAVrh32/33キャプシドをコードする核酸配列を含んでなる核酸分子。
  11. 明細書に開示された発明。
JP2014244953A 2005-04-07 2014-12-03 Aavベクターの機能を上げる方法 Active JP6039632B2 (ja)

Applications Claiming Priority (4)

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

Related Parent Applications (1)

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JP2008505635A Division JP5702519B2 (ja) 2005-04-07 2006-04-07 Aavベクターの機能を上げる方法

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JP2016093699A Division JP6275769B2 (ja) 2005-04-07 2016-05-09 Aavベクターの機能を上げる方法

Publications (3)

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JP2015096070A JP2015096070A (ja) 2015-05-21
JP2015096070A5 true JP2015096070A5 (ja) 2016-01-28
JP6039632B2 JP6039632B2 (ja) 2016-12-07

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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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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

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US (13) US8999678B2 (ja)
EP (12) EP2357010B1 (ja)
JP (5) JP5702519B2 (ja)
CN (6) CN101203613B (ja)
DE (2) DE23193601T1 (ja)
DK (2) DK2359867T3 (ja)
ES (6) ES2525067T3 (ja)
WO (1) WO2006110689A2 (ja)

Families Citing this family (342)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2406745C (en) 2001-11-13 2006-01-10 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
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
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
EP3211085B1 (en) * 2003-09-30 2021-03-31 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
EP2357010B1 (en) * 2005-04-07 2013-06-12 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
ES2400235T3 (es) 2006-04-28 2013-04-08 The Trustees Of The University Of Pennsylvania Método de producción escalable de AAV
ES2535877T3 (es) * 2006-06-19 2015-05-18 Asklepios Biopharmaceutical, Inc. Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica
EP2623605B1 (en) 2007-04-09 2018-11-28 University of Florida Research Foundation, Inc. RAAV vector compositions having tyrosine-modified capsid proteins and methods for use
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
CA2762118A1 (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
ES2724122T3 (es) 2009-04-30 2019-09-06 Univ Pennsylvania Composiciones para dirigir células de las vías respiratorias de conducción que comprenden construcciones de virus adenoasociado
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel 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
BR112012024934A2 (pt) 2010-03-29 2016-12-06 Univ Pennsylvania sistemas de ablação de transgene induzida farmacologicamente
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US9546369B2 (en) 2010-04-23 2017-01-17 University Of Massachusetts Multicistronic expression constructs
EP2826860B1 (en) 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
US9272053B2 (en) 2010-04-23 2016-03-01 University Of Massachusetts AAV-based treatment of cholesterol-related disorders
CA2805170C (en) 2010-07-12 2021-09-21 Fatima Bosch Tubert 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
EP3699286A1 (en) 2011-04-20 2020-08-26 The Trustees of the University of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
EP3318635A1 (en) 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
KR102121355B1 (ko) 2011-04-22 2020-06-11 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 지니는 아데노-관련 바이러스 및 이의 사용 방법
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
BR112014020325A2 (pt) 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
BR122021020419B1 (pt) * 2012-04-18 2023-01-17 The Children's Hospital Of Philadelphia Vetor de vírus adeno-associado (aav) que compreende uma proteína de capsídeo vp1, composições farmacêuticas compreendendo o mesmo, e seus usos
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
TWI775096B (zh) 2012-05-15 2022-08-21 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)
CA2880653C (en) 2012-08-01 2022-05-17 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
CN111621507A (zh) * 2012-09-28 2020-09-04 北卡罗来纳-查佩尔山大学 靶向少突胶质细胞的aav载体
CA2901328C (en) 2013-03-15 2023-08-01 The Trustees Of The University Of Pennsylvania Compositions and methods for treating mps1
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
SG10201709552XA (en) * 2013-05-21 2018-01-30 Univ Florida Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer
JP6600624B2 (ja) * 2013-05-31 2019-10-30 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 アデノ随伴ウイルス変異体及びその使用方法
US9840719B2 (en) * 2013-07-22 2017-12-12 The Children's Hospital Of Philadelphia Variant AAV and compositions, methods and uses for gene transfer to cells, organs and tissues
WO2015048534A1 (en) 2013-09-26 2015-04-02 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
EP3744730A1 (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
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
HUE051311T2 (hu) 2014-03-09 2021-03-01 Univ Pennsylvania Ornitin transzkarbamiláz (TC) deficiencia kezelésében alkalmas készítmények
CN106414474B (zh) 2014-03-17 2021-01-15 阿德夫拉姆生物技术股份有限公司 用于视锥细胞中增强的基因表达的组合物和方法
DK3119797T3 (da) 2014-03-18 2021-03-15 Univ Massachusetts Raav-baserede sammensætninger og fremgangsmåder til behandling af amyotrofisk lateralsklerose
CA2946392A1 (en) 2014-04-25 2015-10-29 James M. Wilson 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
SI3137497T1 (sl) * 2014-05-02 2021-08-31 Genzyme Corporation Vektorji AAV za gensko terapijo mrežnice in centralnega živčnega sistema
JP6974943B2 (ja) 2014-05-13 2021-12-01 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 2種の抗体構築物を発現するaavを含む組成物およびこの使用
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
SG11201701033QA (en) * 2014-08-13 2017-03-30 Philadelphia Children Hospital 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
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
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
KR20170096998A (ko) 2014-11-05 2017-08-25 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 aadc 폴리뉴클레오티드
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
CN112410338A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
RU2727015C2 (ru) 2014-11-21 2020-07-17 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Векторы aav, нацеленные на центральную нервную систему
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
CA2967468A1 (en) 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
EP3242945B1 (en) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
WO2016126857A1 (en) 2015-02-03 2016-08-11 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
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
EP3265178A4 (en) 2015-03-02 2018-09-05 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
DK3268024T3 (da) 2015-03-10 2020-12-07 Univ Columbia Rekombinante glut1-adeno-associerede virusvektorkonstrukter og relaterede fremgangsmåder til genoprettelse af glut1-ekspression
MX2017012097A (es) 2015-03-24 2018-06-15 Univ California Variantes de virus adenoasociado y métodos de uso de estas.
US10555994B2 (en) * 2015-03-30 2020-02-11 Boehringer Ingelheim Animal Health USA Inc. PCV2 ORF2 carrier platform
EP3288594B1 (en) 2015-04-27 2022-06-29 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 METHODS OF DELIVERING AN AGENT TO THE EYE
EP3307310A2 (en) 2015-05-13 2018-04-18 The Trustees Of The University Of Pennsylvania Aav-mediated expression of anti-influenza antibodies and methods of use thereof
US10799566B2 (en) 2015-06-23 2020-10-13 The Children's Hospital Of Philadelphia Modified factor IX, and compositions, methods and uses for gene transfer to cells, organs, and tissues
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
JP7409773B2 (ja) 2015-07-31 2024-01-09 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 改変された細胞および治療の方法
WO2017024198A1 (en) 2015-08-06 2017-02-09 The Trustees Of The University Of Pennsylvania Glp-1 and use thereof in compositions for treating metabolic 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
EP3344650A4 (en) 2015-08-31 2019-04-17 The Trustees Of The University Of Pennsylvania AAV-EPO FOR THE TREATMENT OF PETS
EP3359663A4 (en) 2015-09-24 2019-08-14 The Trustees of The University of Pennsylvania COMPOSITION AND METHOD FOR THE TREATMENT OF A COMPLEMENT-MEDIATED DISEASE
US11273227B2 (en) 2015-10-09 2022-03-15 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
EP3364997B1 (en) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
US20200181644A1 (en) * 2015-10-22 2020-06-11 University Of Florida Research Foundation, Incorporated Synthetic combinatorial aav3 capsid library
CA3002980A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
KR20180100304A (ko) 2015-10-28 2018-09-10 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 유전자 요법을 위한 아데노-관련-바이러스 벡터의 척추강 내 투여
US10983110B2 (en) 2015-12-02 2021-04-20 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
KR20180097631A (ko) 2015-12-11 2018-08-31 매사추세츠 아이 앤드 이어 인퍼머리 핵산을 와우 및 전정 세포에 전달하기 위한 물질 및 방법
ES2918998T3 (es) 2015-12-11 2022-07-21 Univ Pennsylvania Método de purificación escalable para AAVrh10
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
MA43570A (fr) 2015-12-11 2018-11-14 Univ Pennsylvania Thérapie génique pour traiter l'hypercholestérolémie familiale
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
JP7061067B2 (ja) 2015-12-14 2022-04-27 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 クリグラー・ナジャー症候群の処置のための組成物
ES2826384T3 (es) 2015-12-14 2021-05-18 Univ Pennsylvania Terapia génica para trastornos oculares
MX2018007237A (es) * 2015-12-14 2018-12-11 Univ North Carolina Chapel Hill Proteinas de cápside modificadas para un suministro mejorado de vectores de parvovirus.
GB2545763A (en) * 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
CA3011939A1 (en) * 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
WO2017136500A1 (en) 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
EP3443001A4 (en) 2016-04-11 2020-04-29 Obsidian Therapeutics, Inc. REGULATED BIOCIRCUIT SYSTEMS
SG10201912761UA (en) 2016-04-15 2020-02-27 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
KR102574810B1 (ko) 2016-04-15 2023-09-08 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 습성 연령 관련 황반 변성의 치료를 위한 조성물
WO2017181105A1 (en) 2016-04-15 2017-10-19 University Of Massachusetts Methods and compositions for treating metabolic imbalance
JP7140683B2 (ja) 2016-04-15 2022-09-21 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 新規なaav8変異カプシド及びそれを含有する組成物
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
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR THE TREATMENT OF DISEASES
NZ748395A (en) 2016-05-13 2019-12-20 4D Molecular Therapeutics Inc Adeno-associated virus variant capsids and methods of use thereof
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
US11504435B2 (en) 2016-06-13 2022-11-22 The University Of North Carolina At Chapel Hill Optimized CLN1 genes and expression cassettes and their use
WO2017218852A1 (en) 2016-06-15 2017-12-21 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
BR112019000171A2 (pt) 2016-07-08 2019-10-01 Univ Pennsylvania métodos e composições para tratamento de distúrbios e doenças envolvendo rdh12
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
JP6994018B2 (ja) * 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド 新規アデノ随伴ウイルスキャプシドタンパク質
KR102511979B1 (ko) 2016-07-29 2023-03-20 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법
AU2017321488B2 (en) 2016-08-30 2022-10-20 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
GB2605883B (en) 2016-10-18 2023-03-15 Univ 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
JP7065852B2 (ja) 2016-12-01 2022-05-12 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 網膜変性疾患の処置のための医薬組成物
EP3575398A4 (en) * 2017-01-30 2020-11-11 Nippon Medical School Foundation ADENO-ASSOCIATED VIRUS (AAV) CAPSIDE PROTEIN MUTANT
US11554147B2 (en) 2017-02-20 2023-01-17 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
EP3585883A4 (en) 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated PROTEINS OF MODIFIED AAV CAPSIDES AND THEIR USES
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
JP7455579B2 (ja) 2017-02-28 2024-03-26 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 新規アデノ随伴ウイルス(aav)クレードfベクター及びその用途
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
EP3589738A4 (en) 2017-03-01 2021-01-06 The Trustees of The University of Pennsylvania GENE THERAPY FOR EYE DISORDERS
UY37679A (es) 2017-04-14 2018-11-30 Regenxbio Inc Tratamiento de mucopolisacaridosis ii con iduronato-2-sulfatasa (ids) humana recombinante producida por células neurales o gliales humanas
US11879133B2 (en) 2017-04-24 2024-01-23 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CA3061368A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
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
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
CA3098592A1 (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 DISORDERS
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
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
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
CA3071978A1 (en) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CN111163811A (zh) * 2017-08-25 2020-05-15 奥维德医疗公司 重组腺相关载体
KR20200039617A (ko) 2017-08-28 2020-04-16 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 아데노-연관 바이러스 캡시드 변이체 및 이의 사용 방법
JP6825167B2 (ja) 2017-09-20 2021-02-03 4ディー モレキュラー セラピューティクス インコーポレイテッド アデノ随伴ウイルス変異キャプシドおよびその使用方法
SG11202002457RA (en) 2017-09-22 2020-04-29 Univ Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
SG11202003479TA (en) 2017-10-18 2020-05-28 Regenxbio Inc Fully-human post-translationally modified antibody therapeutics
MX2020003945A (es) 2017-10-18 2020-11-09 Regenxbio Inc Tratamiento de enfermedades oculares y cancer de colon metastasico con vegf-trap humano modificado post-traduccionalmente .
BR112020010674A2 (pt) 2017-11-27 2020-11-10 4D Molecular Therapeutics Inc. capsídeos de variante de vírus adeno-associado e uso para inibir a angiogênese
WO2019108856A1 (en) 2017-11-30 2019-06-06 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis iiib
AU2018375164A1 (en) 2017-11-30 2020-06-04 The Trustees Of The University Of Pennsylvania Gene therapy for Mucopolysaccharidosis IIIA
SG11202005618WA (en) 2017-12-19 2020-07-29 Akouos Inc Aav-mediated delivery of therapeutic antibodies to the inner ear
US10806802B2 (en) 2018-01-18 2020-10-20 University Of Guelph Adeno-associated virus particle with mutated capsid and methods of use thereof
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RECOVERING F8 GENE FUNCTION AND METHODS OF USE THEREOF
CA3091806A1 (en) * 2018-02-27 2019-09-06 The Trustees Of The University Of Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
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.
WO2019191418A1 (en) * 2018-03-29 2019-10-03 Asklepios Biopharmaceutical, Inc. Liver tropic recombinant aav6 vectors that evade neutralization
CA3093347A1 (en) 2018-04-05 2019-10-10 Genethon Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism
EP3788165A1 (en) 2018-04-29 2021-03-10 REGENXBIO Inc. 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
CA3098871A1 (en) 2018-05-08 2019-11-14 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
US20210198691A1 (en) 2018-05-15 2021-07-01 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
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
US20220403001A1 (en) 2018-06-12 2022-12-22 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
US20210301305A1 (en) 2018-06-13 2021-09-30 Voyager Therapeutics, Inc. Engineered untranslated regions (utr) for aav production
CN112469822A (zh) 2018-06-14 2021-03-09 再生生物股份有限公司 用于重组aav生产的阴离子交换色谱
GB201811368D0 (en) 2018-07-11 2018-08-29 Ucb Biopharma Sprl Antibody
SG11202100704PA (en) 2018-07-24 2021-02-25 Voyager Therapeutics Inc Systems and methods for producing gene therapy formulations
US20210163991A1 (en) 2018-08-10 2021-06-03 Regenxbio Inc. Scalable method for recombinant aav production
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
JP7450945B2 (ja) 2018-08-30 2024-03-18 テナヤ セラピューティクス, インコーポレイテッド ミオカルディンおよびascl1を用いた心細胞リプログラミング
CN113383010A (zh) 2018-09-28 2021-09-10 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
CN113438954A (zh) 2018-10-01 2021-09-24 宾夕法尼亚州大学信托人 可用于治疗gm1神经节苷脂病的组合物
US20210348242A1 (en) 2018-10-04 2021-11-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
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
EP3867412A1 (en) 2018-10-15 2021-08-25 REGENXBIO Inc. Method for measuring the infectivity of replication defective viral vectors and viruses
AU2019360937A1 (en) 2018-10-15 2021-05-20 Voyager Therapeutics, Inc. Expression vectors for large-scale production of rAAV in the baculovirus/Sf9 system
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
US20210386788A1 (en) 2018-10-24 2021-12-16 Obsidian Therapeutics, Inc. Er tunable protein regulation
EP4269579A3 (en) 2019-01-04 2024-01-24 Ultragenyx Pharmaceutical Inc. Gene therapy constructs for treating wilson disease
WO2020150556A1 (en) 2019-01-18 2020-07-23 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
EP3914229A4 (en) * 2019-01-23 2022-11-02 University of Florida Research Foundation, Incorporated HIGHLY EFFICIENT TRANSDUCTION AND LATERAL SPREADING IN THE RETINA BY A NOVEL ENHANCED AAV VIRUS WITH RATIONAL DESIGN
WO2020160508A1 (en) 2019-01-31 2020-08-06 Oregon Health & Science University Methods for using transcription-dependent directed evolution of aav capsids
TW202045730A (zh) 2019-02-22 2020-12-16 賓州大學委員會 用於治療grn相關的成年發病型神經系統退化症之重組腺相關病毒
WO2020174369A2 (en) 2019-02-25 2020-09-03 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
BR112021016501A2 (pt) 2019-02-25 2021-10-26 Novartis Ag Composições e métodos para tratar distrofia cristalina de bietti
MX2021010266A (es) 2019-02-26 2021-09-23 Univ Pennsylvania Composiciones utiles en el tratamiento de la enfermedad de krabbe.
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
EP3953483B1 (en) 2019-04-11 2023-10-04 REGENXBIO Inc. Methods of size exclusion chromatography for the characterization of recombinant adeno-associated virus compositions
EP4272817A3 (en) 2019-04-19 2024-01-24 RegenxBio Inc. Adeno-associated virus vector formulations and methods
JP2022530126A (ja) * 2019-04-23 2022-06-27 アンセルム(アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル) 新規アデノ随伴ウイルス(aav)バリアント及び遺伝子治療のためのその使用
MX2021012867A (es) 2019-04-24 2022-03-04 Regenxbio Inc Terapéutica de anticuerpos postraduccionalmente modificados completamente humanos.
WO2020222858A1 (en) * 2019-04-27 2020-11-05 Ocugen, Inc. Adeno-associated virus vector mediated gene therapy for ophthalmic diseases
WO2020223236A1 (en) * 2019-04-29 2020-11-05 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
US20240124889A1 (en) 2019-05-07 2024-04-18 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
EP3997225A1 (en) 2019-07-10 2022-05-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of epilepsy
WO2021007515A1 (en) 2019-07-11 2021-01-14 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with micrornas and other factors
BR112022000724A2 (pt) * 2019-07-15 2022-06-14 Meiragtx Uk Ii Ltd Proteínas de capsídeo de aav modificado para tratamento de doença artrítica
US20220396806A1 (en) 2019-07-26 2022-12-15 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
JP2022544004A (ja) 2019-07-26 2022-10-17 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
EP4021936A1 (en) 2019-08-26 2022-07-06 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
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
CN114555814A (zh) 2019-09-13 2022-05-27 罗特格斯新泽西州立大学 Aav相容的层粘连蛋白-连接子聚合蛋白
EP4038194A1 (en) 2019-10-04 2022-08-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
BR112022006718A2 (pt) 2019-10-07 2022-07-12 Regenxbio Inc Composição farmacêutica e métodos de vetor de vírus adeno-associado
US20220403414A1 (en) * 2019-10-16 2022-12-22 Wuxi Apptec (Shanghai) Co., Ltd. Novel aav variant
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
JP2023503637A (ja) 2019-11-28 2023-01-31 リジェネックスバイオ インコーポレイテッド マイクロジストロフィン遺伝子治療コンストラクト及びその使用
AU2020398904A1 (en) * 2019-12-10 2022-06-30 Homology Medicines, Inc. Adeno-associated virus compositions and methods of use thereof
WO2021119053A1 (en) 2019-12-10 2021-06-17 Shire Human Genetic Therapies, Inc. Adeno associated virus vectors for the treatment of hunter disease
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
IL294625A (en) 2020-01-22 2022-09-01 Regenxbio Inc Treatment of mucopolysaccharidosis i with human fully glycosylated human alpha-l-iduronidase (idua)
BR112022015036A2 (pt) 2020-01-29 2022-10-11 Regenxbio Inc Vírus adenoassociado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav, método para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva)
IL294636A (en) 2020-01-29 2022-09-01 Regenxbio Inc Treatment of mucopolysaccharidosis ii with recombinant human idurant-2-sulfatase (ids) produced by human neurons or glial cells
WO2021155337A1 (en) 2020-02-02 2021-08-05 The Trustees Of The University Of Pennsylvania Compositions useful for treating gm1 gangliosidosis
AU2021218703A1 (en) 2020-02-14 2022-07-28 Ultragenyx Pharmaceutical Inc. Gene therapy for treating CDKL5 deficiency disorder
CN115836081A (zh) * 2020-02-25 2023-03-21 儿童医学研究院 腺相关病毒衣壳多肽及载体
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
CA3175034A1 (en) 2020-03-27 2021-09-30 UCB Biopharma SRL Autonomous knob domain peptides
JP2023520402A (ja) 2020-03-31 2023-05-17 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 プロピオン酸血症を処置するための遺伝子治療
WO2021211629A2 (en) * 2020-04-13 2021-10-21 Massachusetts Eye And Ear Infirmary Methods of making and using a vaccine against coronavirus
AU2021257848A1 (en) 2020-04-15 2022-12-01 Voyager Therapeutics, Inc. Tau binding compounds
CA3177407A1 (en) 2020-05-12 2021-11-18 James M. Wilson Compositions for drg-specific reduction of transgene expression
KR20230023637A (ko) 2020-05-12 2023-02-17 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 크라베병의 치료에 유용한 조성물
WO2021231538A2 (en) 2020-05-13 2021-11-18 Akouos, Inc. Compositions and methods for treating kcnq4-associated hearing loss
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
EP4158037A1 (en) 2020-05-26 2023-04-05 Shape Therapeutics Inc. High throughput engineering of functional aav capsids
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
CA3183153A1 (en) 2020-06-17 2021-12-23 Christian HINDERER Compositions and methods for treatment of gene therapy patients
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
CN116438311A (zh) 2020-07-13 2023-07-14 宾夕法尼亚州大学信托人 可用于治疗夏科-马里-图思病的组合物
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
CN111876432B (zh) * 2020-07-29 2022-10-28 舒泰神(北京)生物制药股份有限公司 一组肝靶向新型腺相关病毒的获得及其应用
GB2599212A (en) 2020-07-30 2022-03-30 Shape Therapeutics Inc Stable cell lines for inducible production of rAAV virions
EP4200429A1 (en) 2020-08-24 2023-06-28 The Trustees of The University of Pennsylvania Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
CN116887867A (zh) 2020-08-26 2023-10-13 宾夕法尼亚州大学信托人 用于治疗grn相关成人发作性神经退行性病变的重组腺相关病毒
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
KR20230083287A (ko) 2020-10-07 2023-06-09 리젠엑스바이오 인크. Cln2 질환의 안구 징후에 대한 유전자 요법
TW202228649A (zh) 2020-10-07 2022-08-01 美商銳進科斯生物股份有限公司 諸如形成聚集體之調配物之用於脈絡膜上投與之調配物
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
WO2022076595A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations for suprachoroidal administration such as gel formulations
TW202228648A (zh) 2020-10-07 2022-08-01 美商銳進科斯生物股份有限公司 諸如高黏度調配物之用於脈絡膜上投與之調配物
CA3193697A1 (en) 2020-10-07 2022-04-14 Joseph Bruder Adeno-associated viruses for ocular delivery of gene therapy
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
EP4225906A1 (en) 2020-10-09 2023-08-16 The Trustees of The University of Pennsylvania Compositions and methods for treatment of fabry disease
AR123838A1 (es) 2020-10-18 2023-01-18 Univ Pennsylvania Vector mejorado de virus adenoasociado (aav) y usos de este
EP4236999A1 (en) 2020-10-28 2023-09-06 RegenxBio Inc. Vectorized anti-tnf-alfa antibodies for ocular indications
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
WO2022094255A2 (en) 2020-10-29 2022-05-05 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
JP2023548145A (ja) 2020-10-29 2023-11-15 リジェネックスバイオ インコーポレイテッド 眼の適応症に対するベクター化tnf-アルファアンタゴニスト
CA3200192A1 (en) 2020-12-01 2022-06-09 Justin PERCIVAL Compositions and uses thereof for treatment of angelman syndrome
EP4255570A1 (en) 2020-12-01 2023-10-11 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
CA3200014A1 (en) 2020-12-01 2022-06-09 James M. Wilson Novel compositions with tissue-specific targeting motifs and compositions containing same
US20240167054A1 (en) 2020-12-16 2024-05-23 Regenxbio Inc. Method of producing a recombinant virus particle
WO2022147087A1 (en) 2020-12-29 2022-07-07 Regenxbio Inc. Tau-specific antibody gene therapy compositions, methods and uses thereof
WO2022146839A1 (en) 2020-12-29 2022-07-07 Akouos, Inc. Compositions and methods for treating clrn1-associated hearing loss and/or vision loss
CA3205209A1 (en) 2021-01-21 2022-07-28 Regenxbio Inc. Improved production of recombinant polypeptides and viruses
JP2024505257A (ja) 2021-02-01 2024-02-05 レジェンクスバイオ インコーポレーテッド 神経セロイドリポフスチン症の遺伝子治療
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
BR112023016075A2 (pt) 2021-02-10 2023-11-21 Regenxbio Inc Método para tratar hepatoesplenomegalia, método de distribuição de um raav que codifica hids, método para tratar um sujeito humano, método de tratamento de um sujeito humano, método para determinar a eficácia do tratamento, método para identificar um sujeito como tendo mps ii
JP2024509792A (ja) 2021-02-26 2024-03-05 武田薬品工業株式会社 ファブリー病の治療のための組成物及び方法
EP4323520A1 (en) 2021-04-12 2024-02-21 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
BR112023021971A2 (pt) 2021-04-23 2024-02-20 Univ Pennsylvania Composições com motivos de direcionamento específicos do cérebro e composições contendo os mesmos
JP2024517143A (ja) 2021-04-26 2024-04-19 リジェネックスバイオ インコーポレイテッド ジストロフィン異常症の治療のためのマイクロジストロフィン遺伝子療法投与
EP4334454A2 (en) 2021-05-04 2024-03-13 RegenxBio Inc. Novel aav vectors and methods and uses thereof
WO2022241030A1 (en) 2021-05-11 2022-11-17 Regenxbio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
US20230038295A1 (en) 2021-06-25 2023-02-09 Homology Medicines, Inc. Adeno-associated virus packaging systems
KR20240051112A (ko) 2021-07-01 2024-04-19 인답타 세라뷰틱스 인코포레이티드 조작된 자연 살해(nk) 세포 및 관련 방법
EP4373947A1 (en) 2021-07-19 2024-05-29 New York University Auf1 combination therapies for treatment of muscle degenerative disease
AU2022325955A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce instant blood mediated inflammatory reactions
AU2022326565A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy
WO2023019168A1 (en) 2021-08-11 2023-02-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
IL310691A (en) 2021-08-11 2024-04-01 Sana Biotechnology Inc Genetically modified primary cells for allogeneic cell therapy
AU2022325231A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions
WO2023028510A1 (en) 2021-08-24 2023-03-02 Homology Medicines, Inc. Adeno-associated virus formulations
CA3230004A1 (en) * 2021-08-25 2023-03-02 Yunxiang Zhu Aav particles comprising liver-tropic capsid protein and acid alpha-glucosidase and use to treat pompe disease
IL311572A (en) 2021-09-30 2024-05-01 Akouos Inc Compositions and methods for treating KCNQ4-related hearing loss
WO2023056399A1 (en) 2021-10-02 2023-04-06 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
WO2023060272A2 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
WO2023060269A1 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
WO2023077092A1 (en) 2021-10-28 2023-05-04 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2023077086A1 (en) 2021-10-29 2023-05-04 Homology Medicines, Inc. 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
WO2023102079A1 (en) 2021-12-01 2023-06-08 Shape Therapeutics Inc. Functional aav capsids for systemic administration
WO2023102078A2 (en) 2021-12-01 2023-06-08 Shape Therapeutics Inc. Functional aav capsids for intravitreal administration
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
WO2023106256A1 (ja) * 2021-12-06 2023-06-15 学校法人順天堂 改変型アデノ随伴ウイルスベクター
WO2023114901A2 (en) 2021-12-15 2023-06-22 Oxford Biomedica Solutions Llc Methods and compositions for the production of adeno-associated virus
WO2023133584A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
WO2023133574A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful for treatment of c9orf72-mediated disorders
WO2023133595A2 (en) 2022-01-10 2023-07-13 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
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
WO2023150647A1 (en) 2022-02-02 2023-08-10 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
TW202342525A (zh) 2022-02-02 2023-11-01 美商阿科奧斯公司 用於治療前庭神經鞘瘤相關症狀之抗vegf抗體構築體及相關方法
WO2023158836A1 (en) 2022-02-17 2023-08-24 Sana Biotechnology, Inc. Engineered cd47 proteins and uses thereof
WO2023172491A1 (en) 2022-03-07 2023-09-14 Ultragenyx Pharmaceutical Inc. Modified batch aav production systems and methods
WO2023173123A1 (en) 2022-03-11 2023-09-14 Sana Biotechnology, Inc. Genetically modified cells and compositions and uses thereof
WO2023178053A1 (en) 2022-03-13 2023-09-21 Regenxbio Inc. Modified muscle-specific promoters
WO2023178220A1 (en) 2022-03-16 2023-09-21 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2023183623A1 (en) 2022-03-25 2023-09-28 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
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
WO2023196873A1 (en) 2022-04-06 2023-10-12 Regenxbio Inc. Pharmaceutical composition comprising a recombinant adeno-associated virus vector with an expression cassette encoding a transgene forsuprachoidal administration
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
WO2023196893A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer 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
WO2023201277A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
WO2023201308A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Gene therapy for treating an ocular disease
WO2023215807A1 (en) 2022-05-03 2023-11-09 Regenxbio Inc. VECTORIZED ANTI-TNF-α INHIBITORS FOR OCULAR INDICATIONS
TW202400803A (zh) 2022-05-03 2024-01-01 美商銳進科斯生物股份有限公司 載體化抗補體抗體與補體劑及其投與
WO2023214346A1 (en) 2022-05-06 2023-11-09 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
WO2024007020A1 (en) 2022-06-30 2024-01-04 Indapta Therapeutics, Inc. Combination of engineered natural killer (nk) cells and antibody therapy and related methods
WO2024017990A1 (en) 2022-07-21 2024-01-25 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

Family Cites Families (72)

* 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
FI86885C (fi) 1984-04-20 1992-10-26 Genentech Inc Foerfarande foer framstaellning av human rekombinantfaktor viii och nukleinsyrasekvenser och vektorer anvaend daertill
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
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
US5451521A (en) 1986-05-29 1995-09-19 Genetics Institute, Inc. Procoagulant proteins
US5422260A (en) 1986-05-29 1995-06-06 Genetics Institute, Inc. -Legal Affairs Human factor VIII:c muteins
US5149637A (en) 1987-04-06 1992-09-22 Scripps Clinic & Research Foundation Recombinant Factor VIIIC fragments
IE69026B1 (en) 1987-06-12 1996-08-07 Immuno Ag Novel 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
AU656311B2 (en) 1989-11-17 1995-02-02 Chiron Corporation 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
US5661008A (en) 1991-03-15 1997-08-26 Kabi Pharmacia Ab Recombinant human factor VIII derivatives
SE468050C (sv) 1991-03-15 1998-02-11 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
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
WO1994011013A1 (en) 1992-11-13 1994-05-26 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
ES2226029T3 (es) 1993-06-10 2005-03-16 Bayer Corporation Vector y linea celular de mamifero con productividad mejorada.
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
IL128780A0 (en) 1996-09-06 2000-01-31 Univ Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, 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
EP1015619A1 (en) 1997-09-19 2000-07-05 The Trustees Of The University Of Pennsylvania 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
CA2329060C (en) 1998-05-28 2011-09-13 John A. Chiorini 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
EP1127150B1 (en) 1998-11-05 2007-05-16 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6491907B1 (en) 1998-11-10 2002-12-10 The University Of North Carolina At Chapel Hill Recombinant parvovirus vectors and method of making
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
US20020045264A1 (en) * 2000-03-14 2002-04-18 During Matthew J. Production of chimeric capsid vectors
WO2001074164A1 (en) * 2000-03-31 2001-10-11 The General Hospital Corporation Methods of modulating hair growth
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
CA2453405A1 (en) * 2001-07-13 2003-01-23 University Of Iowa Research Foundation Pseudotyped adeno-associated 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
WO2003042361A2 (en) * 2001-11-09 2003-05-22 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
CA2406745C (en) * 2001-11-13 2006-01-10 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
ATE348153T1 (de) * 2002-04-29 2007-01-15 Univ Pennsylvania Methode für die direkte gewinnung und amplifikation von integrierten viren aus zellulärer gewebe-dna
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
EP3211085B1 (en) 2003-09-30 2021-03-31 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
EP1828390B1 (en) * 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
EP2357010B1 (en) * 2005-04-07 2013-06-12 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
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
ES2400235T3 (es) 2006-04-28 2013-04-08 The Trustees Of The University Of Pennsylvania Método de producción escalable de AAV
JP2009535339A (ja) 2006-04-28 2009-10-01 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 低下されたキャプシド免疫原性を有する改変aavベクターおよびその使用
US20120322861A1 (en) * 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
BR122021020419B1 (pt) * 2012-04-18 2023-01-17 The Children's Hospital Of Philadelphia Vetor de vírus adeno-associado (aav) que compreende uma proteína de capsídeo vp1, composições farmacêuticas compreendendo o mesmo, e seus usos
US9840719B2 (en) * 2013-07-22 2017-12-12 The Children's Hospital Of Philadelphia Variant AAV and compositions, methods and uses for gene transfer to cells, organs and tissues
EP3744730A1 (en) * 2013-10-11 2020-12-02 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
US10799566B2 (en) 2015-06-23 2020-10-13 The Children's Hospital Of Philadelphia Modified factor IX, and compositions, methods and uses for gene transfer to cells, organs, and tissues
LT3872085T (lt) * 2015-07-30 2023-05-10 Massachusetts Eye & Ear Infirmary Viruso prototipo sekos ir jų panaudojimas
TW202208397A (zh) * 2020-05-13 2022-03-01 美商航海家醫療公司 Aav蛋白殼之趨性重定向

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