JP2017506521A5 - - Google Patents

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JP2017506521A5
JP2017506521A5 JP2016569022A JP2016569022A JP2017506521A5 JP 2017506521 A5 JP2017506521 A5 JP 2017506521A5 JP 2016569022 A JP2016569022 A JP 2016569022A JP 2016569022 A JP2016569022 A JP 2016569022A JP 2017506521 A5 JP2017506521 A5 JP 2017506521A5
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capsid protein
recombinant aav
aav vector
aav2 capsid
tissue
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JP2016569022A
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JP6602788B2 (ja
JP2017506521A (ja
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Priority to JP2019186201A priority Critical patent/JP6942166B2/ja
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JP2016569022A 2014-02-17 2015-02-17 アデノ随伴ウイルスベクター Active JP6602788B2 (ja)

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JP2019186201A JP6942166B2 (ja) 2014-02-17 2019-10-09 アデノ随伴ウイルスベクター

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US201461940639P 2014-02-17 2014-02-17
US61/940,639 2014-02-17
GB1403684.2 2014-03-03
GBGB1403684.2A GB201403684D0 (en) 2014-03-03 2014-03-03 Vector
PCT/EP2015/053335 WO2015121501A1 (en) 2014-02-17 2015-02-17 Adeno-associated virus vector

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JP2017506521A JP2017506521A (ja) 2017-03-09
JP2017506521A5 true JP2017506521A5 (enExample) 2018-03-29
JP6602788B2 JP6602788B2 (ja) 2019-11-06

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JP2016569022A Active JP6602788B2 (ja) 2014-02-17 2015-02-17 アデノ随伴ウイルスベクター
JP2019186201A Active JP6942166B2 (ja) 2014-02-17 2019-10-09 アデノ随伴ウイルスベクター
JP2021086867A Active JP7309780B2 (ja) 2014-02-17 2021-05-24 アデノ随伴ウイルスベクター
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JP2021086867A Active JP7309780B2 (ja) 2014-02-17 2021-05-24 アデノ随伴ウイルスベクター
JP2023109822A Active JP7612767B2 (ja) 2014-02-17 2023-07-04 アデノ随伴ウイルスベクター

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US (4) US20180135076A1 (enExample)
EP (2) EP3108000B1 (enExample)
JP (4) JP6602788B2 (enExample)
KR (1) KR102082032B1 (enExample)
CN (1) CN106062200B (enExample)
AU (2) AU2015216847B2 (enExample)
BR (1) BR112016018598A2 (enExample)
CA (2) CA3126061C (enExample)
DK (1) DK3108000T3 (enExample)
ES (1) ES2749235T3 (enExample)
GB (1) GB201403684D0 (enExample)
HU (1) HUE045801T2 (enExample)
IL (2) IL247332B (enExample)
MX (1) MX372992B (enExample)
PL (1) PL3108000T3 (enExample)
PT (1) PT3108000T (enExample)
RU (1) RU2743382C2 (enExample)
SG (1) SG11201606365TA (enExample)
SI (1) SI3108000T1 (enExample)
WO (1) WO2015121501A1 (enExample)

Families Citing this family (203)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
CA2833905C (en) 2010-04-23 2019-09-10 University Of Massachusetts Multicistronic expression constructs
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
ES2704677T3 (es) 2013-04-18 2019-03-19 Fond Telethon Suministro eficaz de genes grandes por vectores de AAV duales
CA2907799A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
WO2015048534A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
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
AU2015231294B2 (en) 2014-03-18 2020-10-29 University Of Massachusetts rAAV-based compositions and methods for treating amyotrophic lateral sclerosis
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
WO2015168666A2 (en) * 2014-05-02 2015-11-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
EP3795580A1 (en) 2014-10-03 2021-03-24 University of Massachusetts High efficiency library-identified aav vectors
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
CN112553229A (zh) 2014-11-05 2021-03-26 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
RU2020108189A (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (als)
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
US20180057839A1 (en) * 2014-11-26 2018-03-01 The Regents Of The University Of California Therapeutic compositions comprising transcription factors and methods of making and using the same
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
CA3021949C (en) 2015-04-24 2023-10-17 University Of Massachusetts Modified aav constructs and uses thereof
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
JP7064214B2 (ja) * 2015-09-28 2022-05-10 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 抗体回避性ウイルスベクターのための方法および組成物
WO2017070525A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
US11426469B2 (en) 2015-10-22 2022-08-30 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
GB201519086D0 (en) * 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
PE20231949A1 (es) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
RU2021102893A (ru) 2015-11-05 2021-03-03 Бамбу Терапьютикс, Инк. Модифицированные гены атаксии фридрейха и векторы для генной терапии
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
DK3387137T3 (da) * 2015-12-11 2021-05-03 California Inst Of Techn Målretningspeptider til styring af adeno-associerede virusser (aav`er)
WO2017106354A1 (en) * 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Adeno-associated viral vectors useful in treatment of spinal muscular atropy
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
EP3413928B1 (en) 2016-02-12 2022-04-20 University of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
WO2017147477A1 (en) * 2016-02-26 2017-08-31 University Of Florida Research Foundation, Inc. Aav heparin mutants that display significantly improved eye and brain transduction
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
CN107287238B (zh) * 2016-04-11 2020-10-16 厦门继景生物技术有限责任公司 一种基因载体及其用于治疗雷柏氏先天性黑矇2型疾病的基因治疗药物
IL322403A (en) 2016-04-15 2025-09-01 Univ Pennsylvania Preparations for the treatment of wet age-related macular degeneration
WO2017181105A1 (en) 2016-04-15 2017-10-19 University Of Massachusetts Methods and compositions for treating metabolic imbalance
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
BR112018073472A2 (pt) 2016-05-18 2019-08-27 Voyager Therapeutics, Inc. composições e métodos de tratamento da doença de huntington
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2017218852A1 (en) 2016-06-15 2017-12-21 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
MA44546B1 (fr) * 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
RU2753194C2 (ru) 2016-06-21 2021-08-12 Бамбу Терапьютикс, Инк. Оптимизированные гены и экспрессионные кассеты мини-дистрофина и их применение
CA3029119A1 (en) 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of friedreich ataxia and other related disorders
US11427838B2 (en) 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
US11174469B2 (en) 2016-06-29 2021-11-16 Crispr Therapeutics Ag Materials and methods for treatment of Amyotrophic Lateral Sclerosis (ALS) and other related disorders
CN109843914B (zh) 2016-07-06 2024-03-15 沃泰克斯药物股份有限公司 用于治疗疼痛相关病症的材料和方法
US11801313B2 (en) 2016-07-06 2023-10-31 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
WO2018011572A1 (en) 2016-07-12 2018-01-18 The University Of Manchester Gene therapy
CA3029833A1 (en) 2016-07-29 2018-02-01 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
JP7226801B2 (ja) * 2016-08-16 2023-02-21 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 標的遺伝子導入のための方法および組成物
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
EP3518985A4 (en) 2016-09-29 2020-08-05 University of Florida Research Foundation, Incorporated AAVRH.10 VARIANTS WITH HOST ANTIBODY EXHAUST CAPACITIES AND MODIFIED TISSUE TARGETING PROPERTIES
US11578340B2 (en) 2016-10-13 2023-02-14 University Of Massachusetts AAV capsid designs
AU2017345470B2 (en) * 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
IL266264B2 (en) 2016-11-04 2023-11-01 Baxalta Inc Adeno-associated virus formulations
KR20250022877A (ko) 2017-01-31 2025-02-17 리젠엑스바이오 인크. 완전히-인간 글리코실화된 인간 알파-l-이두로니다아제(idua)를 이용한 뮤코다당증 1형의 치료
JP7237843B2 (ja) 2017-02-21 2023-03-13 ユニバーシティー オブ フロリダ リサーチ ファンデーション, インク. 修飾されたaavキャプシドタンパク質およびその使用
US20200040061A1 (en) 2017-02-22 2020-02-06 Crispr Therapeutics Ag Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders
US11920148B2 (en) 2017-02-22 2024-03-05 Crispr Therapeutics Ag Compositions and methods for gene editing
WO2018154439A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of spinocerebellar ataxia type 1 (sca1) and other spinocerebellar ataxia type 1 protein (atxn1) gene related conditions or disorders
US11407997B2 (en) 2017-02-22 2022-08-09 Crispr Therapeutics Ag Materials and methods for treatment of primary hyperoxaluria type 1 (PH1) and other alanine-glyoxylate aminotransferase (AGXT) gene related conditions or disorders
WO2018154462A2 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of spinocerebellar ataxia type 2 (sca2) and other spinocerebellar ataxia type 2 protein (atxn2) gene related conditions or disorders
WO2018191666A1 (en) 2017-04-14 2018-10-18 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2 sulfatase (ids) produced by human neural or glial cells
CN111108198A (zh) 2017-05-05 2020-05-05 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
EP3622073A4 (en) 2017-05-09 2021-01-06 University of Massachusetts METHOD FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
CN110831611A (zh) * 2017-05-10 2020-02-21 马萨诸塞眼科耳科诊所 用于修饰病毒的组装活化蛋白(aap)依赖性的方法和组合物
CA3061968A1 (en) 2017-05-10 2018-11-15 Massachusetts Eye And Ear Infirmary Methods and compositions for modifying assembly-activating protein (aap)-dependence of viruses
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
BR112019019015A2 (pt) 2017-06-30 2020-04-14 Univ California vírions de vírus adeno-associado com capsídeos variantes e seus métodos de uso
CN111132626B (zh) 2017-07-17 2024-01-30 沃雅戈治疗公司 轨迹阵列引导系统
WO2019028306A2 (en) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES
WO2019040586A1 (en) * 2017-08-25 2019-02-28 Ovid Therapeutics Inc. RECOMBINANT ADENO-ASSOCIATE 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
JP7397488B2 (ja) 2017-09-22 2023-12-13 ユニバーシティ オブ マサチューセッツ Sod1二重発現ベクターおよびその使用
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
CN111479924B (zh) 2017-10-16 2024-06-14 沃雅戈治疗公司 肌萎缩性侧索硬化症(als)的治疗
CN111727201A (zh) * 2017-10-18 2020-09-29 再生生物股份有限公司 完全人源翻译后修饰的抗体治疗剂
WO2019123430A1 (en) 2017-12-21 2019-06-27 Casebia Therapeutics Llp Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp)
AU2018393050A1 (en) 2017-12-21 2020-06-18 Bayer Healthcare Llc Materials and methods for treatment of Usher Syndrome Type 2A
US12558434B2 (en) 2018-02-20 2026-02-24 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
WO2019178412A1 (en) * 2018-03-16 2019-09-19 Research Institute At Nationwide Children's Hospital Increasing tissue specific gene delivery by capsid modification
CN112218880A (zh) * 2018-03-29 2021-01-12 阿斯克肋匹奥生物制药公司 逃避中和的肝趋向性重组aav6载体
US12091435B2 (en) 2018-04-03 2024-09-17 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
JP7425738B2 (ja) 2018-04-03 2024-01-31 ギンコ バイオワークス インコーポレイテッド 眼組織を標的とするウイルスベクター
MX2020010464A (es) 2018-04-03 2021-01-29 Vectores de virus que evitan anticuerpos.
CN108660159B (zh) * 2018-04-12 2020-10-30 四川大学 重组蝙蝠腺相关病毒载体及其用途
KR102051393B1 (ko) 2018-05-04 2019-12-04 (주)지뉴인텍 아데노부속바이러스 기반 유전자 전달용 재조합 벡터 및 이를 이용한 암질환 예방 또는 치료용 조성물
AU2019270900B2 (en) 2018-05-15 2026-03-12 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties
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
AU2019268330A1 (en) 2018-05-15 2020-11-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
US12163129B2 (en) 2018-06-08 2024-12-10 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
WO2020014479A1 (en) 2018-07-11 2020-01-16 Baxalta Incorporated Aav compositions
EA202190358A1 (ru) * 2018-07-31 2021-05-04 Корнелл Юниверсити Способы генной терапии для контроля функции органов
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CN119684417A (zh) 2018-10-02 2025-03-25 沃雅戈治疗公司 重新定向aav衣壳的嗜性
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
WO2020102645A1 (en) * 2018-11-16 2020-05-22 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease
GB201900702D0 (en) 2019-01-18 2019-03-06 Univ Bristol Therapy
EP3911410A1 (en) 2019-01-18 2021-11-24 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
CN113728108A (zh) * 2019-02-15 2021-11-30 桑格摩生物治疗股份有限公司 用于生产重组aav的组合物和方法
JP7698583B2 (ja) 2019-03-21 2025-06-25 ギンコ バイオワークス インコーポレイテッド 組換えアデノ随伴ウイルスベクター
KR20210142725A (ko) * 2019-03-28 2021-11-25 오피노 엘엘씨 코어 페이징 처리
SG11202110607WA (en) 2019-04-01 2021-10-28 Tenaya Therapeutics Inc Adeno-associated virus with engineered capsid
CA3136872A1 (en) * 2019-04-23 2020-10-29 Inserm (Institut National De La Sante Et De La Recherche Medicale) New adeno-associated virus (aav) variants and uses thereof for gene therapy
AU2020263757B2 (en) 2019-04-24 2025-08-28 Takara Bio Inc. AAV mutant having brain-targeting property
CN113767110A (zh) * 2019-04-26 2021-12-07 北卡罗来纳-查佩尔山大学 用于双重聚糖结合aav2.5载体的方法和组合物
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
CA3144864A1 (en) 2019-06-27 2020-12-30 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
US12173290B2 (en) 2019-06-28 2024-12-24 Crispr Therapeutics Ag Materials and methods for controlling gene editing
US20220372512A1 (en) * 2019-07-04 2022-11-24 Children's Medical Research Institute Methods and aav vectors for in vivo transduction
WO2021009805A1 (ja) * 2019-07-12 2021-01-21 株式会社遺伝子治療研究所 ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン
EP3999119B1 (en) * 2019-07-15 2024-09-11 Meiragtx UK II Limited Modified aav capsid proteins for treatment of arthritic disease
WO2021030764A1 (en) * 2019-08-14 2021-02-18 University Of Florida Research Foundation, Incorporated Aav capsid variants for gene therapy
JP2022545534A (ja) * 2019-08-29 2022-10-27 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー ヒト血液脳関門を通過するためのアデノ随伴ウイルスベクター
US20220333133A1 (en) 2019-09-03 2022-10-20 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
EP4041755A4 (en) * 2019-10-10 2023-10-25 Solid Biosciences Inc. Modified aav capsids and uses thereof
EP4045664A1 (en) * 2019-10-16 2022-08-24 Wuxi Apptec (Shanghai) Co., Ltd. A novel aav variant
IL292297A (en) 2019-10-17 2022-06-01 Stridebio Inc Adeno-associated viral vectors for treatment of niemann-pick disease type c
BR112022014852A2 (pt) * 2020-01-29 2022-10-11 Genzyme Corp Proteínas modificadas do capsídeo viral adenoassociado para terapia gênica ocular e métodos de uso das mesmas
EP4135841A1 (en) 2020-04-15 2023-02-22 Voyager Therapeutics, Inc. Tau binding compounds
IL298001A (en) 2020-05-13 2023-01-01 Voyager Therapeutics Inc Redirecting tropism of adeno-associated virus capsids
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
GB202010009D0 (en) * 2020-06-30 2020-08-12 Syncona Investment Man Ltd Vector
WO2022026409A1 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
CN111825772B (zh) * 2020-07-30 2023-10-20 中国科学院精密测量科学与技术创新研究院 具有变异衣壳蛋白的腺相关病毒及其应用
CA3181024A1 (en) 2020-08-06 2022-02-10 Fundacion Para La Investigacion Medica Aplicada Viral particles for use in treating tauopathies such as alzheimer's diseases by gene therapy
BR112023002374A2 (pt) 2020-08-12 2023-03-21 UCB Biopharma SRL Terapia genética usando construções de ácido nucléico compreendendo sequências promotoras de proteína 2 de ligação de metil cpg (mecp2)
CA3189878A1 (en) 2020-08-19 2022-02-24 Colin O'BANION Adeno-associated virus vectors for treatment of rett syndrome
JP7811576B2 (ja) * 2020-08-28 2026-02-05 ジョイント・ストック・カンパニー “バイオキャド” SARS-CoV-2に対するAAV5に基づくワクチン
CN113754728B (zh) * 2020-09-30 2022-07-12 广州派真生物技术有限公司 腺相关病毒突变体及其应用
MX2023003699A (es) * 2020-10-07 2023-04-21 Regenxbio Inc Virus adenoasociados para el suministro ocular de genoterapia.
CN115698304A (zh) 2020-10-09 2023-02-03 特纳亚治疗股份有限公司 亲斑蛋白-2基因疗法的方法和组合物
CN116390935A (zh) 2020-10-09 2023-07-04 Ucb生物制药有限责任公司 核酸构建体、病毒载体和病毒颗粒
CN116490514A (zh) 2020-10-28 2023-07-25 北卡罗来纳-查佩尔山大学 用于双重聚糖结合aav2.5载体的方法和组合物
WO2022093769A1 (en) * 2020-10-28 2022-05-05 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
EP4240861A1 (en) 2020-11-03 2023-09-13 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
AU2021378075B2 (en) 2020-11-11 2025-03-27 Borea Therapeutics S.R.L. Modified viral particles for gene therapy
WO2022130172A1 (en) 2020-12-15 2022-06-23 Pfizer Inc. Hilic uplc-ms method for separating and analyzing intact adeno-associated virus capsid proteins
US20240011048A1 (en) 2020-12-21 2024-01-11 Pfizer Inc. Methods and compositions for inhibiting excess nucleic acid precipitation
AU2021404944A1 (en) 2020-12-23 2023-07-06 Pfizer Inc. Methods for purification of aav vectors by affinity chromatography
WO2022173605A2 (en) 2021-02-10 2022-08-18 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
GB202101958D0 (en) 2021-02-12 2021-03-31 Ucl Business Ltd Gene therapy for dopamine transporter deficiency syndrome
CN112961219B (zh) * 2021-02-24 2022-08-12 中国农业科学院生物技术研究所 重组腺相关病毒、其突变体及其构建方法和应用
CN115044614B (zh) * 2021-03-09 2023-10-20 上海目镜生物医药科技有限公司 一种用于基因靶向与表达的aav-8型血清型的改造型载体及其构建方法及应用
US20240307553A1 (en) 2021-04-01 2024-09-19 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
JP2024515902A (ja) 2021-04-26 2024-04-10 アレクシオン ファーマ インターナショナル オペレーションズ リミテッド 組織向性が改善されたアデノ随伴ウイルスベクターカプシド
CN117355342A (zh) * 2021-05-07 2024-01-05 奥伊斯特普安生物制药公司 编码神经营养因子的腺相关病毒病毒颗粒和其用途
KR20240005950A (ko) 2021-05-12 2024-01-12 폰다지오네 텔레톤 이티에스 벡터 시스템
EP4359547A1 (en) 2021-06-22 2024-05-01 Pfizer Inc. Production of adeno-associated virus vector in insect cells
WO2023283962A1 (en) 2021-07-16 2023-01-19 Huigene Therapeutics Co., Ltd. Modified aav capsid for gene therapy and methods thereof
CA3234809A1 (en) 2021-10-20 2023-04-27 Steven Goldman Isolated glial progenitor cells for use in the competition treatment of age-related white matter loss
MX2024005171A (es) 2021-10-28 2024-05-13 UCB Biopharma SRL Constructos de acido nucleico, vectores viricos y particulas viricas.
WO2023081648A1 (en) 2021-11-02 2023-05-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
EP4426331A1 (en) 2021-11-02 2024-09-11 University of Rochester Tcf7l2 mediated remyelination in the brain
EP4442830A4 (en) 2021-11-29 2025-04-30 Shanghai Regenelead Therapies Co., Ltd. AADC/GDNF polynucleotide and use thereof in the treatment of Parkinson's disease
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
AU2022423978A1 (en) * 2021-12-28 2024-07-18 Chengdu Origen Biotechnology Co., Ltd. Modified aav capsid protein and use thereof
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
AU2023220237A1 (en) 2022-02-21 2024-08-15 Shanghai Regenelead Therapies Co., Ltd Vegf-binding molecule and pharmaceutical use thereof
JP2025507751A (ja) 2022-03-01 2025-03-21 クリスパー・セラピューティクス・アクチェンゲゼルシャフト アンジオポエチン様3(angptl3)関連状態を処置するための方法および組成物
CA3255225A1 (en) 2022-04-04 2023-10-12 The Regents Of The University Of California COMPOSITIONS AND METHODS OF GENETIC COMPLEMENTATION
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
CA3255627A1 (en) 2022-05-06 2023-11-09 Novartis Ag NEW VP2 FUSION POLYPEPTIDES OF RECOMBINANT AAV
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
AR129843A1 (es) * 2022-07-06 2024-10-02 Voyager Therapeutics Inc Variantes de la cápside de aav y usos de estas
US20260041792A1 (en) * 2022-07-12 2026-02-12 University Of Florida Research Foundation, Incorporated Novel aav3b capsid variants with enhanced hepatocyte tropism
KR20240014846A (ko) * 2022-07-26 2024-02-02 숙명여자대학교산학협력단 Aav2-anks1a 재조합 바이러스를 포함하는 알츠하이머 질병 치료용 조성물
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
CN116063404B (zh) * 2022-09-27 2025-08-29 广州译码基因科技有限公司 可提高AAV包装能力的衣壳蛋白MutD及其应用
GB202216168D0 (en) 2022-10-31 2022-12-14 UCB Biopharma SRL Route of administration
CN116041443B (zh) * 2022-12-30 2023-09-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN115925819B (zh) * 2022-12-30 2023-10-13 广州派真生物技术有限公司 腺相关病毒突变体及其应用
EP4646485A1 (en) * 2023-01-05 2025-11-12 Emugen Therapeutics LLC Chimeric aav and uses thereof
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
EP4658294A2 (en) 2023-02-02 2025-12-10 University of Rochester Competitive replacement of glial cells
WO2024189094A1 (en) 2023-03-14 2024-09-19 UCB Biopharma SRL Gene therapy
GB202304767D0 (en) * 2023-03-30 2023-05-17 Ucl Business Ltd Synthetic AAV Capsid
WO2024238853A1 (en) * 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2025022290A1 (en) 2023-07-21 2025-01-30 Crispr Therapeutics Ag Modulating expression of alas1 (5'-aminolevulinate synthase 1) gene
WO2025049917A1 (en) * 2023-08-31 2025-03-06 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
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
CN118440161B (zh) * 2024-01-16 2025-02-25 沈阳兴齐眼药股份有限公司 提高aav病毒视网膜穿透能力的衣壳蛋白突变体及其应用
WO2025186726A1 (en) 2024-03-05 2025-09-12 Crispr Therapeutics Ag Modulating expression of agt (angiotensinogen) gene
WO2025228976A1 (en) 2024-04-29 2025-11-06 UCB Biopharma SRL Gene therapy
GB202407038D0 (en) 2024-05-17 2024-07-03 Axovia Therapeutics Ltd Novel Gene Therapy
WO2025250454A1 (en) 2024-05-28 2025-12-04 University Of Rochester Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo
WO2025262203A1 (en) 2024-06-21 2025-12-26 UCB Biopharma SRL Nucleic acid sequences
WO2026017965A1 (en) 2024-07-19 2026-01-22 The University Court Of The University Of Edinburgh Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions

Family Cites Families (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5639872A (en) 1993-07-27 1997-06-17 Hybridon, Inc. Human VEGF-specific oligonucleotides
AU1686997A (en) 1995-12-08 1997-06-27 Hybridon, Inc. Modified vegf antisense oligonucleotides for treatment of skin disorders
EP0932694A2 (en) 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
DE69939169D1 (de) 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
PT1127150E (pt) 1998-11-05 2007-08-22 Univ Pennsylvania ''sequências de ácido nucleico do vírus adeno associado do serotipo 1, vectores e células hospedeiras que as contêm''
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
US20030144221A1 (en) 2001-07-17 2003-07-31 Isis Pharmaceuticals Inc. Antisense modulation of BCL2-associated X protein expression
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
CN103555677B (zh) 2001-11-13 2018-01-30 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
WO2003052051A2 (en) 2001-12-17 2003-06-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
PT2573170T (pt) 2001-12-17 2018-03-26 Univ Pennsylvania Sequências de um vírus adenoassociado (aav) de serotipo 9, vetor contendo as mesmas, e suas utilizações
EP1456383B1 (en) 2001-12-21 2014-03-12 Medigene AG A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism
EP1496944B1 (en) 2002-05-01 2008-08-20 University of Florida Research Foundation, Inc. Improved raav expression systems for genetic modification of specific capsid proteins
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
JP2007524386A (ja) 2003-06-19 2007-08-30 アビジェン, インコーポレイテッド 減少した免疫反応性を有するaavビリオン、およびその使用
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
DK3211085T3 (da) * 2003-09-30 2021-06-21 Univ Pennsylvania Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf
WO2005089224A2 (en) 2004-03-12 2005-09-29 Alnylam Pharmaceuticals, Inc. iRNA AGENTS TARGETING VEGF
US7892809B2 (en) 2004-12-15 2011-02-22 The University Of North Carolina At Chapel Hill Chimeric vectors
US8999678B2 (en) * 2005-04-07 2015-04-07 The Trustees Of The University Of Pennsylvania Method of increasing the function of an AAV vector
US7543116B2 (en) 2006-01-30 2009-06-02 International Business Machines Corporation Data processing system, cache system and method for handling a flush operation in a data processing system having multiple coherency domains
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US20090317417A1 (en) 2006-04-28 2009-12-24 The Trustees Of The University Of Pennsylvania Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
ES2385679T3 (es) 2006-08-24 2012-07-30 Virovek, Inc. Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos
US7872118B2 (en) 2006-09-08 2011-01-18 Opko Ophthalmics, Llc siRNA and methods of manufacture
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
US20090215879A1 (en) 2008-02-26 2009-08-27 University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
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
US9133479B2 (en) 2009-06-03 2015-09-15 Cedars-Sinai Medical Center Effective vector platform for gene transfer and gene therapy
CN102474479B (zh) 2009-07-22 2016-01-20 阿瓦尔有限公司 改进的数据包检测器
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
CN102071206A (zh) * 2010-10-22 2011-05-25 中山大学 一种腺相关病毒衣壳蛋白基因、相应的蛋白及其应用
DK4005603T3 (da) * 2011-04-22 2024-11-25 Univ California Adeno-associerede virus-virioner med variant kapsid og fremgangsmåder til anvendelse heraf
US9169299B2 (en) 2011-08-24 2015-10-27 The Board Of Trustees Of The Leleand Stanford Junior University AAV capsid proteins for nucleic acid transfer
RU2683497C2 (ru) * 2012-04-18 2019-03-28 Дзе Чилдрен'З Хоспитал Оф Филадельфия Композиция и способы высокоэффективного переноса генов с помощью вариантов капсида aav
WO2013170078A1 (en) 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
TWI702955B (zh) 2012-05-15 2020-09-01 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)
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
WO2014186746A1 (en) 2013-05-16 2014-11-20 University Of Florida Research Foundation, Inc. HAIRPIN mRNA ELEMENTS AND METHODS FOR THE REGULATION OF PROTEIN TRANSLATION
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
US10308957B2 (en) 2014-03-04 2019-06-04 University Of Florida Research Foundation, Inc. rAAV vectors and methods for transduction of photoreceptors and RPE cells
WO2016133917A1 (en) 2015-02-16 2016-08-25 University Of Florida Research Foundation Raav vector compositions, methods for targeting vascular endothelial cells and use in treatment of type i diabetes
JP7064214B2 (ja) 2015-09-28 2022-05-10 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 抗体回避性ウイルスベクターのための方法および組成物
WO2017066764A2 (en) 2015-10-16 2017-04-20 William Marsh Rice University Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
WO2018011572A1 (en) 2016-07-12 2018-01-18 The University Of Manchester Gene therapy

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