JPWO2021003442A5 - - Google Patents
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- Publication number
- JPWO2021003442A5 JPWO2021003442A5 JP2022500533A JP2022500533A JPWO2021003442A5 JP WO2021003442 A5 JPWO2021003442 A5 JP WO2021003442A5 JP 2022500533 A JP2022500533 A JP 2022500533A JP 2022500533 A JP2022500533 A JP 2022500533A JP WO2021003442 A5 JPWO2021003442 A5 JP WO2021003442A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- vector
- lsd
- lysosomal enzyme
- optionally
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| 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 | ||
| PCT/US2020/040770 WO2021003442A1 (en) | 2019-07-02 | 2020-07-02 | Vector compositions and methods of using same for treatment of lysosomal storage disorders |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022538497A JP2022538497A (ja) | 2022-09-02 |
| JP2022538497A5 JP2022538497A5 (https=) | 2023-07-13 |
| JPWO2021003442A5 true JPWO2021003442A5 (https=) | 2023-07-13 |
Family
ID=72046993
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022500533A Pending JP2022538497A (ja) | 2019-07-02 | 2020-07-02 | ライソゾーム蓄積症を処置するためのベクター組成物およびそれを使用する方法 |
Country Status (10)
| Country | Link |
|---|---|
| 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=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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)
| 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 |
-
2020
- 2020-07-02 US US17/624,196 patent/US12492412B2/en active Active
- 2020-07-02 CA CA3145662A patent/CA3145662A1/en active Pending
- 2020-07-02 BR BR112021026866A patent/BR112021026866A2/pt unknown
- 2020-07-02 AU AU2020298575A patent/AU2020298575A1/en active Pending
- 2020-07-02 EP EP20754414.9A patent/EP3994253A1/en active Pending
- 2020-07-02 TW TW109122470A patent/TW202122579A/zh unknown
- 2020-07-02 JP JP2022500533A patent/JP2022538497A/ja active Pending
- 2020-07-02 CN CN202080061537.2A patent/CN114616000A/zh active Pending
- 2020-07-02 WO PCT/US2020/040770 patent/WO2021003442A1/en not_active Ceased
- 2020-07-02 KR KR1020227002362A patent/KR20220069917A/ko active Pending
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