JP2022530833A5 - - Google Patents
Info
- Publication number
- JP2022530833A5 JP2022530833A5 JP2021565001A JP2021565001A JP2022530833A5 JP 2022530833 A5 JP2022530833 A5 JP 2022530833A5 JP 2021565001 A JP2021565001 A JP 2021565001A JP 2021565001 A JP2021565001 A JP 2021565001A JP 2022530833 A5 JP2022530833 A5 JP 2022530833A5
- Authority
- JP
- Japan
Links
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962840911P | 2019-04-30 | 2019-04-30 | |
| US62/840,911 | 2019-04-30 | ||
| US201962913401P | 2019-10-10 | 2019-10-10 | |
| US62/913,401 | 2019-10-10 | ||
| PCT/US2020/030484 WO2020223356A1 (en) | 2019-04-30 | 2020-04-29 | Compositions useful for treatment of pompe disease |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022530833A JP2022530833A (ja) | 2022-07-01 |
| JPWO2020223356A5 JPWO2020223356A5 (https=) | 2023-05-08 |
| JP2022530833A5 true JP2022530833A5 (https=) | 2023-05-08 |
| JP7585233B2 JP7585233B2 (ja) | 2024-11-18 |
Family
ID=73029176
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021564730A Pending JP2022530824A (ja) | 2019-04-30 | 2020-04-29 | ポンペ病の治療のために有用な組成物 |
| JP2021565001A Active JP7585233B2 (ja) | 2019-04-30 | 2020-04-29 | ポンペ病の治療のために有用な組成物 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021564730A Pending JP2022530824A (ja) | 2019-04-30 | 2020-04-29 | ポンペ病の治療のために有用な組成物 |
Country Status (16)
| Country | Link |
|---|---|
| US (2) | US20220193207A1 (https=) |
| EP (2) | EP3980548A4 (https=) |
| JP (2) | JP2022530824A (https=) |
| KR (2) | KR20220004696A (https=) |
| CN (2) | CN114127275A (https=) |
| AU (2) | AU2020266829A1 (https=) |
| BR (2) | BR112021021792A2 (https=) |
| CA (2) | CA3134523A1 (https=) |
| CL (2) | CL2021002754A1 (https=) |
| CO (2) | CO2021016198A2 (https=) |
| IL (2) | IL287522A (https=) |
| MX (2) | MX2021013364A (https=) |
| PH (2) | PH12021552638A1 (https=) |
| SG (2) | SG11202111400TA (https=) |
| TW (1) | TW202100541A (https=) |
| WO (2) | WO2020223362A1 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FI3635009T3 (fi) | 2017-06-07 | 2026-04-07 | Regeneron Pharma | Koostumuksia ja menetelmiä entsyymien internalisoimiseksi |
| JP7779653B2 (ja) | 2018-02-07 | 2025-12-03 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 治療用タンパク質送達のための方法および組成物 |
| EP3793591A1 (en) | 2018-05-17 | 2021-03-24 | Regeneron Pharmaceuticals, Inc. | Anti-cd63 antibodies, conjugates, and uses thereof |
| CN115087458A (zh) * | 2019-10-10 | 2022-09-20 | 阿米库斯治疗学公司 | 变体igf2构建体 |
| KR20230010255A (ko) * | 2020-05-14 | 2023-01-18 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 폼페병의 치료에 유용한 조성물 |
| US20230220069A1 (en) | 2020-06-17 | 2023-07-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of gene therapy patients |
| EP4430201A4 (en) * | 2021-11-12 | 2026-04-08 | Univ Pennsylvania | Gene therapy for the treatment of mucopolysaccharidosis IIIA |
| NL2031676B1 (en) * | 2022-04-22 | 2023-11-07 | Univ Erasmus Med Ct Rotterdam | Gene therapy for Pompe Disease |
| WO2024003687A1 (en) * | 2022-06-28 | 2024-01-04 | Pfizer Inc. | Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy |
| WO2024151982A1 (en) * | 2023-01-13 | 2024-07-18 | Amicus Therapeutics, Inc. | Gene therapy constructs for the treatment of pompe disease |
| CN116693633B (zh) * | 2023-02-21 | 2023-12-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2025171163A1 (en) * | 2024-02-06 | 2025-08-14 | Duke University | Compositions for and methods of treating lysosomal storage diseases |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ATE521701T1 (de) * | 2003-01-22 | 2011-09-15 | Univ Duke | Verbesserte konstrukte zur expression lysosomaler polypeptide |
| CN1922313B (zh) * | 2004-02-10 | 2011-10-26 | 生物马林医药公司 | 酸性α-糖苷酶及其片段 |
| US20090117091A1 (en) * | 2006-11-13 | 2009-05-07 | Lebowitz Jonathan | Methods for treating pompe disease |
| EP2623599B1 (en) * | 2007-10-04 | 2019-01-02 | Roche Innovation Center Copenhagen A/S | Micromirs |
| HRP20170698T1 (hr) * | 2008-05-07 | 2017-09-22 | Biomarin Pharmaceutical Inc. | Lizosomski usmjereni peptidi i njihove uporabe |
| US8466340B2 (en) * | 2009-02-18 | 2013-06-18 | Amicus Therapeutics, Inc. | Mouse model for pompe disease and methods of use thereof |
| JP5755569B2 (ja) * | 2009-02-25 | 2015-07-29 | セファイド | 肺癌を検出する方法 |
| EP2475376B1 (en) * | 2009-06-17 | 2016-03-30 | BioMarin Pharmaceutical Inc. | Formulations for lysosomal enzymes |
| US9279007B2 (en) | 2010-11-22 | 2016-03-08 | Amicus Therapeutics, Inc. | Signal sequences to improve protein expressions and secretion of recombinant enzymes and other proteins |
| WO2012070014A2 (en) * | 2010-11-26 | 2012-05-31 | Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. | Identification of novel cell surface markers for pancreatic progenitor cells and definite endodermal cells |
| GB201103062D0 (en) * | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| LT2714752T (lt) * | 2011-05-27 | 2018-03-12 | Amicus Therapeutics, Inc. | Nukreipiančių peptidų kopuliavimo ant rekombinantinių lizosominių fermentų būdai, skirti lizosominio kaupimo ligų gydymo pagerinimui |
| WO2013013019A2 (en) * | 2011-07-21 | 2013-01-24 | Alnylam Pharmaceuticals, Inc. | Lysosomal polypeptides, methods of making and using |
| CN107405411A (zh) * | 2014-05-01 | 2017-11-28 | 华盛顿大学 | 使用腺病毒载体的体内基因改造 |
| KR102423442B1 (ko) * | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| IL325155A (en) * | 2016-03-30 | 2026-02-01 | Amicus Therapeutics Inc | Formulations containing recombinant acid alpha-glucosidase |
| EP3293259A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3293260A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| MX2019002842A (es) * | 2016-09-12 | 2019-08-29 | Genethon | Variantes de alfa-glucosidasa acida y usos de las mismas. |
| CA3057899A1 (en) * | 2017-03-27 | 2018-10-04 | Vrije Universiteit Brussel | Diaphragm-specific nucleic acid regulatory elements and methods and use thereof |
| JP2018198611A (ja) * | 2018-08-03 | 2018-12-20 | ウニベルシダッド アウトノマ デ バルセロナ | 脂肪組織に形質導入するのに有用なアデノ随伴ウイルスベクター |
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2020
- 2020-04-29 CN CN202080048355.1A patent/CN114127275A/zh active Pending
- 2020-04-29 TW TW109114314A patent/TW202100541A/zh unknown
- 2020-04-29 JP JP2021564730A patent/JP2022530824A/ja active Pending
- 2020-04-29 WO PCT/US2020/030493 patent/WO2020223362A1/en not_active Ceased
- 2020-04-29 MX MX2021013364A patent/MX2021013364A/es unknown
- 2020-04-29 PH PH1/2021/552638A patent/PH12021552638A1/en unknown
- 2020-04-29 SG SG11202111400TA patent/SG11202111400TA/en unknown
- 2020-04-29 WO PCT/US2020/030484 patent/WO2020223356A1/en not_active Ceased
- 2020-04-29 BR BR112021021792A patent/BR112021021792A2/pt unknown
- 2020-04-29 KR KR1020217038231A patent/KR20220004696A/ko not_active Withdrawn
- 2020-04-29 US US17/606,410 patent/US20220193207A1/en not_active Abandoned
- 2020-04-29 CN CN202080049015.0A patent/CN114072515A/zh active Pending
- 2020-04-29 EP EP20798851.0A patent/EP3980548A4/en not_active Withdrawn
- 2020-04-29 SG SG11202111380VA patent/SG11202111380VA/en unknown
- 2020-04-29 JP JP2021565001A patent/JP7585233B2/ja active Active
- 2020-04-29 CA CA3134523A patent/CA3134523A1/en active Pending
- 2020-04-29 EP EP20799541.6A patent/EP3963063A4/en not_active Withdrawn
- 2020-04-29 PH PH1/2021/552637A patent/PH12021552637A1/en unknown
- 2020-04-29 US US17/606,414 patent/US20220193261A1/en active Pending
- 2020-04-29 AU AU2020266829A patent/AU2020266829A1/en not_active Abandoned
- 2020-04-29 KR KR1020217038223A patent/KR20220008280A/ko not_active Withdrawn
- 2020-04-29 AU AU2020266552A patent/AU2020266552A1/en not_active Abandoned
- 2020-04-29 CA CA3134485A patent/CA3134485A1/en active Pending
- 2020-04-29 BR BR112021021720A patent/BR112021021720A2/pt unknown
- 2020-04-29 MX MX2021013365A patent/MX2021013365A/es unknown
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2021
- 2021-10-20 CL CL2021002754A patent/CL2021002754A1/es unknown
- 2021-10-20 CL CL2021002755A patent/CL2021002755A1/es unknown
- 2021-10-24 IL IL287522A patent/IL287522A/en unknown
- 2021-10-24 IL IL287523A patent/IL287523A/en unknown
- 2021-11-29 CO CONC2021/0016198A patent/CO2021016198A2/es unknown
- 2021-11-29 CO CONC2021/0016200A patent/CO2021016200A2/es unknown