CN111163811A - 重组腺相关载体 - Google Patents

重组腺相关载体 Download PDF

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Publication number
CN111163811A
CN111163811A CN201880064381.6A CN201880064381A CN111163811A CN 111163811 A CN111163811 A CN 111163811A CN 201880064381 A CN201880064381 A CN 201880064381A CN 111163811 A CN111163811 A CN 111163811A
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aav
vector
raavrec3
nucleic acid
vectors
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马修·杜林
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Ovid Therapeutics Inc
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Ovid Therapeutics Inc
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • C—CHEMISTRY; METALLURGY
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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CN201880064381.6A 2017-08-25 2018-08-22 重组腺相关载体 Pending CN111163811A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762550458P 2017-08-25 2017-08-25
US62/550,458 2017-08-25
PCT/US2018/047466 WO2019040586A1 (en) 2017-08-25 2018-08-22 RECOMBINANT ADENO-ASSOCIATE VECTORS

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CN111163811A true CN111163811A (zh) 2020-05-15

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US (2) US20190085358A1 (https=)
EP (1) EP3655041A4 (https=)
JP (1) JP2020533968A (https=)
KR (1) KR20200042935A (https=)
CN (1) CN111163811A (https=)
AU (1) AU2018320849A1 (https=)
CA (1) CA3073937A1 (https=)
IL (1) IL272655A (https=)
MX (1) MX2020002148A (https=)
WO (1) WO2019040586A1 (https=)

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JP2022514194A (ja) 2018-11-21 2022-02-10 セルテゴ セラピューティクス インコーポレイテッド 自殺のリスクを低減し、うつ病を迅速に軽減するためのガボキサドール
CA3149164A1 (en) * 2019-09-03 2021-03-11 Suma Krishnan Compositions and methods for the treatment of congenital ichthyoses
MX2022014599A (es) 2020-05-20 2022-12-16 Certego Therapeutics Inc Gaboxadol deuterado en anillo y su uso para el tratamiento de trastornos psiquiatricos.
CA3191543A1 (en) * 2020-09-04 2022-03-10 Lei Cao Novel engineered capsid serotype of recombinant adeno-associated viral vector with enhanced transduction efficiency and widespread distribution in the brain
US20230086710A1 (en) * 2021-09-14 2023-03-23 California Institute Of Technology Aav capsid variants

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CA2745131A1 (en) * 1998-05-28 1999-12-02 John A. Chiorini Aav5 vector and uses thereof
WO2000075365A2 (en) * 1999-06-08 2000-12-14 University Of Iowa Research Foundation Compounds and methods to enhance raav transduction
US20060018841A1 (en) * 2004-06-03 2006-01-26 Avigen, Inc. AAV vectors for gene delivery to the lung
CN101203613A (zh) * 2005-04-07 2008-06-18 宾夕法尼亚州立大学托管会 增强腺相关病毒载体功能的方法
US20160074474A1 (en) * 2013-05-01 2016-03-17 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
CN105579465A (zh) * 2013-07-22 2016-05-11 费城儿童医院 用于基因转移到细胞、器官和组织中的变异aav和组合物、方法及用途
CN106062200A (zh) * 2014-02-17 2016-10-26 伦敦国王学院 腺相关病毒载体
CN106574256A (zh) * 2014-05-30 2017-04-19 斯坦福大学托管董事会 用于潜伏病毒感染的递送治疗的组合物和方法
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CN109476707A (zh) * 2016-05-13 2019-03-15 4D分子治疗有限公司 腺相关病毒变体衣壳和其使用方法
CN110520536A (zh) * 2017-02-15 2019-11-29 北卡罗来纳-查佩尔山大学 用于穿过维管结构的基因转移的方法和组合物
CN111867595A (zh) * 2017-12-20 2020-10-30 奥维德医疗公司 hM4Di在治疗癫痫发作紊乱中的用途
CN112739353A (zh) * 2018-06-14 2021-04-30 奥维德医疗公司 Mir-92a或mir-145在治疗安格曼综合征中的用途
WO2021113634A1 (en) * 2019-12-05 2021-06-10 The Board Of Regents Of The University Of Texas Transgene cassettes designed to express a human mecp2 gene
TW202132570A (zh) * 2019-11-19 2021-09-01 美商艾司科皮歐斯生物製藥有限公司 用於治療龐貝症及溶酶體病症之包含肝特異性啟動子之治療性腺相關病毒
CN113966399A (zh) * 2018-09-26 2022-01-21 加州理工学院 用于靶向基因疗法的腺相关病毒组合物
CA3116391A1 (en) * 2020-09-14 2022-03-14 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
CN114259502A (zh) * 2021-12-24 2022-04-01 南京鼓楼医院 一种基于脂肪组织的中枢靶向递送siRNA的方法及应用
CN114450393A (zh) * 2019-07-15 2022-05-06 查尔斯河实验室股份有限公司 重组修饰腺相关病毒辅助载体及其在提高重组修饰腺相关病毒的包装效率的用途
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CN111867595A (zh) * 2017-12-20 2020-10-30 奥维德医疗公司 hM4Di在治疗癫痫发作紊乱中的用途
CN112739353A (zh) * 2018-06-14 2021-04-30 奥维德医疗公司 Mir-92a或mir-145在治疗安格曼综合征中的用途
CN113966399A (zh) * 2018-09-26 2022-01-21 加州理工学院 用于靶向基因疗法的腺相关病毒组合物
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WO2021113634A1 (en) * 2019-12-05 2021-06-10 The Board Of Regents Of The University Of Texas Transgene cassettes designed to express a human mecp2 gene
CA3116391A1 (en) * 2020-09-14 2022-03-14 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
WO2022169984A1 (en) * 2021-02-03 2022-08-11 The University Of North Carolina At Chapel Hill Protein m analogs and fusion proteins and their use for inhibiting antibody function
WO2022235702A1 (en) * 2021-05-04 2022-11-10 California Institute Of Technology Recombinant aavs for delivery to central nervous system and brain vasculature
CN114259502A (zh) * 2021-12-24 2022-04-01 南京鼓楼医院 一种基于脂肪组织的中枢靶向递送siRNA的方法及应用
CN115554418A (zh) * 2022-11-22 2023-01-03 四川至善唯新生物科技有限公司 一种重组腺相关病毒载体的药物组合物及其用途

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WO2019040586A1 (en) 2019-02-28
IL272655A (en) 2020-03-31
KR20200042935A (ko) 2020-04-24
AU2018320849A1 (en) 2020-03-05
US20210371880A1 (en) 2021-12-02
JP2020533968A (ja) 2020-11-26
MX2020002148A (es) 2020-07-20
EP3655041A1 (en) 2020-05-27
EP3655041A4 (en) 2021-04-21
US20190085358A1 (en) 2019-03-21
CA3073937A1 (en) 2019-02-28

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