BR112018011881A2 - proteínas capsidiais modificadas para liberação aumentada de vetores de parvovírus - Google Patents

proteínas capsidiais modificadas para liberação aumentada de vetores de parvovírus

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Publication number
BR112018011881A2
BR112018011881A2 BR112018011881-8A BR112018011881A BR112018011881A2 BR 112018011881 A2 BR112018011881 A2 BR 112018011881A2 BR 112018011881 A BR112018011881 A BR 112018011881A BR 112018011881 A2 BR112018011881 A2 BR 112018011881A2
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BR
Brazil
Prior art keywords
capsid proteins
increased release
modified capsid
parvovirus vectors
vectors
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Application number
BR112018011881-8A
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English (en)
Inventor
Jude Samulski Richard
Warischalk Jayme
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The University Of North Carolina At Chapel Hill
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Publication date
Application filed by The University Of North Carolina At Chapel Hill filed Critical The University Of North Carolina At Chapel Hill
Publication of BR112018011881A2 publication Critical patent/BR112018011881A2/pt

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    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
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    • A61K38/162—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
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Abstract

a presente invenção refere-se a proteínas capsidiais de parvovírus modificadas com eficiência de transdução aumentada, vetores virais compreendendo as mesmas, e métodos de utilização das mesmas para liberação de ácidos nucléicos para uma célula ou um sujeito.
BR112018011881-8A 2015-12-14 2016-12-14 proteínas capsidiais modificadas para liberação aumentada de vetores de parvovírus BR112018011881A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562266941P 2015-12-14 2015-12-14
US62/266,941 2015-12-14
PCT/US2016/066466 WO2017106236A1 (en) 2015-12-14 2016-12-14 Modified capsid proteins for enhanced delivery of parvovirus vectors

Publications (1)

Publication Number Publication Date
BR112018011881A2 true BR112018011881A2 (pt) 2018-12-04

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BR112018011881-8A BR112018011881A2 (pt) 2015-12-14 2016-12-14 proteínas capsidiais modificadas para liberação aumentada de vetores de parvovírus

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Country Link
US (2) US11124546B2 (pt)
EP (1) EP3390429A4 (pt)
JP (3) JP7406783B2 (pt)
CN (2) CN108602859B (pt)
AU (2) AU2016371779C1 (pt)
BR (1) BR112018011881A2 (pt)
CA (1) CA3008013A1 (pt)
MX (2) MX2018007237A (pt)
RU (1) RU2751953C2 (pt)
WO (1) WO2017106236A1 (pt)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US10335466B2 (en) 2014-11-05 2019-07-02 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of parkinson's disease
CN112410339A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
JP2017535266A (ja) 2014-11-14 2017-11-30 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 筋萎縮性側索硬化症（ａｌｓ）を治療する組成物および方法
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
IL292830B2 (en) 2015-09-28 2023-06-01 Univ Florida Methods and compositions for stealth virus antibody vectors
WO2017096162A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
JP7220080B2 (ja) 2016-05-18 2023-02-09 ボイジャー セラピューティクス インコーポレイテッド ハンチントン病治療組成物及び方法
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
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3095127A1 (en) * 2018-03-29 2019-10-03 Asklepios Biopharmaceutical, Inc. Liver tropic recombinant aav6 vectors that evade neutralization
CA3094311A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
AU2019270900A1 (en) * 2018-05-15 2020-10-15 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
CA3137106A1 (en) * 2019-04-26 2020-10-29 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
US20220372512A1 (en) * 2019-07-04 2022-11-24 Children's Medical Research Institute Methods and aav vectors for in vivo transduction
CA3157700A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
CN110950934B (zh) * 2019-12-31 2022-11-04 复旦大学 一种腺相关病毒衣壳蛋白、载体及其构建方法与应用
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
JP2023513749A (ja) 2020-02-13 2023-04-03 テナヤ セラピューティクス， インコーポレイテッド 心疾患を治療するための遺伝子療法ベクター
AU2021358957A1 (en) 2020-10-07 2023-06-08 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21
US20240066080A1 (en) * 2020-12-23 2024-02-29 Synteny Therapeutics, Inc. Protoparvovirus and tetraparvovirus compositions and methods for gene therapy
EP4367251A1 (en) 2021-07-08 2024-05-15 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
WO2024054864A1 (en) 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Cardioprotective heart disease therapies

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2385837T3 (es) 2004-12-15 2012-08-01 The University Of North Carolina At Chapel Hill Vectores quiméricos
CN106906241B (zh) 2005-04-07 2020-09-11 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
US20090191597A1 (en) * 2006-01-20 2009-07-30 Asklepios Biopharmaceutical, Inc. Enhanced production of infectious parvovirus vectors in insect cells
WO2007089632A2 (en) * 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
EP2016174A2 (en) 2006-04-28 2009-01-21 The Trustees of the University of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
US8889641B2 (en) * 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
CA2885544A1 (en) * 2012-09-28 2014-04-03 The University Of North Carolina At Chapel Hill Aav vectors targeted to oligodendrocytes
SG10201709552XA (en) 2013-05-21 2018-01-30 Univ Florida Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer
US10081659B2 (en) * 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
IL292830B2 (en) * 2015-09-28 2023-06-01 Univ Florida Methods and compositions for stealth virus antibody vectors

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