BR112020003976A8 - Vírus adeno-associado (aav) com domínio fosfolipase modificado - Google Patents

Vírus adeno-associado (aav) com domínio fosfolipase modificado

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Publication number
BR112020003976A8
BR112020003976A8 BR112020003976A BR112020003976A BR112020003976A8 BR 112020003976 A8 BR112020003976 A8 BR 112020003976A8 BR 112020003976 A BR112020003976 A BR 112020003976A BR 112020003976 A BR112020003976 A BR 112020003976A BR 112020003976 A8 BR112020003976 A8 BR 112020003976A8
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BR
Brazil
Prior art keywords
modified
aav
adeno
associated virus
sequence
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BR112020003976A
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English (en)
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BR112020003976A2 (pt
Inventor
STRINGS-UFOMBAH Vanessa
KAO Shih-Chu
W Roelvink Petrus
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Benitec Biopharma Ltd
Benitec Biopharma Pty Ltd
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Application filed by Benitec Biopharma Ltd, Benitec Biopharma Pty Ltd filed Critical Benitec Biopharma Ltd
Publication of BR112020003976A2 publication Critical patent/BR112020003976A2/pt
Publication of BR112020003976A8 publication Critical patent/BR112020003976A8/pt

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  • Health & Medical Sciences (AREA)
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Abstract

A presente divulgação refere-se geralmente ao vírus adeno-associado (AAV) modificado de sorotipos diferentes do sorotipo 2, que possuem uma proteína de capsídeo viral com uma sequência da subunidade 1 (VP1) que é modificada em relação à sequência do tipo selvagem correspondente. Em particular, os AAVs modificados da divulgação compreendem substituições de aminoácidos específicas de sítio no domínio fosfolipase A2 (PLA2) e sequência de flanqueamento em relação à sequência de tipo selvagem correspondente que melhora a funcionalidade do AAV quando produzido em células de inseto. A presente divulgação também se refere a métodos de produção dos AAVs modificados, reagentes para os mesmos, sistemas de expressão de baculovírus e células de inseto para a produção dos referidos AAVs modificados.
BR112020003976A 2017-08-31 2018-08-30 Vírus adeno-associado (aav) com domínio fosfolipase modificado BR112020003976A8 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762553028P 2017-08-31 2017-08-31
US62/553,028 2017-08-31
PCT/IB2018/056651 WO2019043630A1 (en) 2017-08-31 2018-08-30 ADENO-ASSOCIATED VIRUS (AAV) WITH MODIFIED PHOSPHOLIPASE DOMAIN

Publications (2)

Publication Number Publication Date
BR112020003976A2 BR112020003976A2 (pt) 2020-09-01
BR112020003976A8 true BR112020003976A8 (pt) 2023-02-28

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Country Status (12)

Country Link
US (1) US11499141B2 (pt)
EP (1) EP3676381A4 (pt)
JP (1) JP7360382B2 (pt)
KR (1) KR102612563B1 (pt)
CN (1) CN111183224B (pt)
AU (1) AU2018323049A1 (pt)
BR (1) BR112020003976A8 (pt)
CA (1) CA3074042A1 (pt)
IL (1) IL272972A (pt)
TW (1) TWI802584B (pt)
UY (1) UY37863A (pt)
WO (1) WO2019043630A1 (pt)

Families Citing this family (7)

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Publication number Priority date Publication date Assignee Title
WO2018139634A1 (ja) * 2017-01-30 2018-08-02 学校法人日本医科大学 アデノ随伴ウイルス（ａａｖ）キャプシドタンパク質の変異体
US11499141B2 (en) 2017-08-31 2022-11-15 Benitec IP Holdings Inc. Adeno-associated virus (AAV) with modified phospholipase domain
JP7389040B2 (ja) * 2018-01-17 2023-11-29 メイラジーティーエックス・ユーケー・ザ・セカンド・リミテッド 遺伝子治療のための修飾ｒＡＡＶキャプシドタンパク質
CN113631706A (zh) 2019-02-28 2021-11-09 贝尼泰克知识产权控股股份有限公司 用于治疗眼咽肌营养不良(opmd)的组合物和方法
CN111349148A (zh) * 2020-03-13 2020-06-30 辉大(上海)生物科技有限公司 一种腺相关病毒载体及其用途
WO2021225921A1 (en) * 2020-05-04 2021-11-11 The University Of North Carolina At Chapel Hill Modified adeno-associated virus vectors that evade neutralizing antibodies and uses thereof
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

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WO2005072364A2 (en) 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector
WO2009137006A2 (en) * 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
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
US11499141B2 (en) 2017-08-31 2022-11-15 Benitec IP Holdings Inc. Adeno-associated virus (AAV) with modified phospholipase domain

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KR102612563B1 (ko) 2023-12-11
TWI802584B (zh) 2023-05-21
UY37863A (es) 2019-03-29
EP3676381A1 (en) 2020-07-08
EP3676381A4 (en) 2021-06-02
TW201920676A (zh) 2019-06-01
US11499141B2 (en) 2022-11-15
JP7360382B2 (ja) 2023-10-12
BR112020003976A2 (pt) 2020-09-01
AU2018323049A1 (en) 2020-04-16
CN111183224A (zh) 2020-05-19
KR20200058427A (ko) 2020-05-27
JP2020533973A (ja) 2020-11-26
CN111183224B (zh) 2024-02-23
CA3074042A1 (en) 2019-03-07
US20200190481A1 (en) 2020-06-18
WO2019043630A1 (en) 2019-03-07
IL272972A (en) 2020-04-30

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