JP7585233B2 - ポンペ病の治療のために有用な組成物 - Google Patents
ポンペ病の治療のために有用な組成物 Download PDFInfo
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| JP2023526923A (ja) * | 2020-05-14 | 2023-06-26 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ポンペ病の治療に有用な組成物 |
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| DK3635009T3 (da) | 2017-06-07 | 2026-03-30 | Regeneron Pharma | Sammensætninger og fremgangsmåder til internalisering af enzymer |
| US12258597B2 (en) | 2018-02-07 | 2025-03-25 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for therapeutic protein delivery |
| BR112020023145A2 (pt) | 2018-05-17 | 2021-02-02 | Regeneron Pharmaceuticals, Inc. | anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto |
| KR20220077921A (ko) * | 2019-10-10 | 2022-06-09 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 변이체 igf2 작제물 |
| AU2021292200A1 (en) | 2020-06-17 | 2023-02-02 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of gene therapy patients |
| KR20240133693A (ko) * | 2021-11-12 | 2024-09-04 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 점액다당류증 iiia의 치료를 위한 유전자 요법 |
| 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 |
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| WO2010096369A1 (en) | 2009-02-18 | 2010-08-26 | Amicus Therapeutics, Inc. | Mouse model for pompe disease and methods of use thereof |
| JP2012518421A (ja) | 2009-02-25 | 2012-08-16 | セファイド | 肺癌を検出する方法 |
| JP2014503198A (ja) | 2010-11-22 | 2014-02-13 | カリダス・バイオファーマ，インコーポレーテッド | 組換え酵素および他のタンパク質のタンパク質発現および分泌を改善する新規シグナル配列 |
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