TW202122579A - 用於治療溶酶體儲積症之載體組合物及其使用方法 - Google Patents

用於治療溶酶體儲積症之載體組合物及其使用方法 Download PDF

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TW202122579A
TW202122579A TW109122470A TW109122470A TW202122579A TW 202122579 A TW202122579 A TW 202122579A TW 109122470 A TW109122470 A TW 109122470A TW 109122470 A TW109122470 A TW 109122470A TW 202122579 A TW202122579 A TW 202122579A
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composition
vector
lysosomal
lsd
lysosomal enzyme
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TW109122470A
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Chinese (zh)
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曰強 杜
柳林
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美商Ｍ６Ｐ生物醫藥公司
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TW109122470A 2019-07-02 2020-07-02 用於治療溶酶體儲積症之載體組合物及其使用方法 TW202122579A (zh)

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US201962869808P 2019-07-02 2019-07-02
US201962869781P 2019-07-02 2019-07-02
US62/869,808 2019-07-02
US62/869,781 2019-07-02

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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

Family Cites Families (17)

* 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
US6905856B2 (en) * 2001-12-21 2005-06-14 Genzyme Glycobiology Research Institute, Inc. Soluble GlcNAc phosphotransferase
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
PT2561069T (pt) * 2010-04-23 2017-05-22 Alexion Pharma Inc Enzima de doença de armazenamento lisossomal
US20150030582A1 (en) 2010-04-23 2015-01-29 Synageva Biopharma Corp. Lysosomal Storage Disease Enzyme
US10722559B2 (en) * 2014-08-11 2020-07-28 Shire Human Genetic Therapies, Inc. Mannose-6-phosphate bearing peptides fused to lysosomal enzymes
WO2018064667A1 (en) * 2016-09-30 2018-04-05 Washington University COMPOSITIONS COMPRISING A MODIFIED GlcNAc-1-PHOSPHOTRANSFERASE AND METHODS OF USE THEREOF

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US20220380800A1 (en) 2022-12-01
CN114616000A (zh) 2022-06-10
EP3994253A1 (en) 2022-05-11
US12492412B2 (en) 2025-12-09
KR20220069917A (ko) 2022-05-27
WO2021003442A1 (en) 2021-01-07
JP2022538497A (ja) 2022-09-02
CA3145662A1 (en) 2021-01-07
BR112021026866A2 (pt) 2022-03-03

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