CN114616000A - 载体组合物及其用于治疗溶酶体贮积症的方法 - Google Patents

载体组合物及其用于治疗溶酶体贮积症的方法 Download PDF

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CN114616000A
CN114616000A CN202080061537.2A CN202080061537A CN114616000A CN 114616000 A CN114616000 A CN 114616000A CN 202080061537 A CN202080061537 A CN 202080061537A CN 114616000 A CN114616000 A CN 114616000A
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composition
vector
lysosomal
lysosomal enzyme
lsd
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C·杜
L·刘
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M6p Medical Switzerland Co ltd
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CN202080061537.2A 2019-07-02 2020-07-02 载体组合物及其用于治疗溶酶体贮积症的方法 Pending CN114616000A (zh)

Applications Claiming Priority (5)

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US201962869808P 2019-07-02 2019-07-02
US201962869781P 2019-07-02 2019-07-02
US62/869808 2019-07-02
US62/869781 2019-07-02
PCT/US2020/040770 WO2021003442A1 (en) 2019-07-02 2020-07-02 Vector compositions and methods of using same for treatment of lysosomal storage disorders

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CN114616000A true CN114616000A (zh) 2022-06-10

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US (1) US12492412B2 (https=)
EP (1) EP3994253A1 (https=)
JP (1) JP2022538497A (https=)
KR (1) KR20220069917A (https=)
CN (1) CN114616000A (https=)
AU (1) AU2020298575A1 (https=)
BR (1) BR112021026866A2 (https=)
CA (1) CA3145662A1 (https=)
TW (1) TW202122579A (https=)
WO (1) WO2021003442A1 (https=)

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JP2024505885A (ja) * 2021-01-27 2024-02-08 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル リソソーム酸リパーゼ欠損症（ｌａｌ－ｄ）の治療のための材料及び方法
CN113238053B (zh) * 2021-04-30 2022-05-13 四川大学华西医院 一种用于检测stat3二聚化的质粒
WO2023150051A1 (en) * 2022-02-04 2023-08-10 M6P Therapeutics, Inc. Compositions and methods of using two-promoter vector for treatment of lysosomal storage disorders
WO2023150388A1 (en) * 2022-02-07 2023-08-10 M6P Therapeutics, Inc. Compositions comprising glucocerebrocidase and methods of use thereof
WO2025238106A1 (en) 2024-05-15 2025-11-20 M6P Therapeutics (Switzerland) GmbH Lysosome-targeting degradation fusion design
CN119950766A (zh) * 2025-02-11 2025-05-09 中国医学科学院阜外医院 一种治疗pnpla2突变致心律失常性心肌病的基因药物及其制备方法和应用

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030119088A1 (en) * 2001-12-21 2003-06-26 Novazyme Pharmaceuticals, Inc. Soluble GlcNAc phosphotransferase
CN102985538A (zh) * 2010-04-23 2013-03-20 辛那杰瓦生物制药股份有限公司 溶酶体贮积病酶
CN106573039A (zh) * 2014-08-11 2017-04-19 夏尔人类遗传性治疗公司 与溶酶体酶融合的载有甘露糖‑6‑磷酸的肽
WO2018064667A1 (en) * 2016-09-30 2018-04-05 Washington University COMPOSITIONS COMPRISING A MODIFIED GlcNAc-1-PHOSPHOTRANSFERASE AND METHODS OF USE THEREOF

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5585362A (en) 1989-08-22 1996-12-17 The Regents Of The University Of Michigan Adenovirus vectors for gene therapy
US5350674A (en) 1992-09-04 1994-09-27 Becton, Dickinson And Company Intrinsic factor - horse peroxidase conjugates and a method for increasing the stability thereof
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
CA2386270A1 (en) 1999-10-15 2001-04-26 University Of Massachusetts Rna interference pathway genes as tools for targeted genetic interference
US6326193B1 (en) 1999-11-05 2001-12-04 Cambria Biosciences, Llc Insect control agent
AU2001275474A1 (en) 2000-06-12 2001-12-24 Akkadix Corporation Materials and methods for the control of nematodes
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
CN1856576B (zh) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
US20150030582A1 (en) 2010-04-23 2015-01-29 Synageva Biopharma Corp. Lysosomal Storage Disease Enzyme

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030119088A1 (en) * 2001-12-21 2003-06-26 Novazyme Pharmaceuticals, Inc. Soluble GlcNAc phosphotransferase
CN102985538A (zh) * 2010-04-23 2013-03-20 辛那杰瓦生物制药股份有限公司 溶酶体贮积病酶
CN106573039A (zh) * 2014-08-11 2017-04-19 夏尔人类遗传性治疗公司 与溶酶体酶融合的载有甘露糖‑6‑磷酸的肽
WO2018064667A1 (en) * 2016-09-30 2018-04-05 Washington University COMPOSITIONS COMPRISING A MODIFIED GlcNAc-1-PHOSPHOTRANSFERASE AND METHODS OF USE THEREOF

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
LIN LIU等: "Engineering of GlcNAc-1-Phosphotransferase for Production of Highly Phosphorylated Lysosomal Enzymes for Enzyme Replacement Therapy", MOL THER METHODS CLIN DEV, vol. 5, pages 59 - 65, XP055667960, DOI: 10.1016/j.omtm.2017.03.006 *
郑振宇等: "基因工程", 31 March 2015, 华中科技大学出版社, pages: 251 - 252 *

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