JP2023503046A5 - - Google Patents
Info
- Publication number
- JP2023503046A5 JP2023503046A5 JP2022529007A JP2022529007A JP2023503046A5 JP 2023503046 A5 JP2023503046 A5 JP 2023503046A5 JP 2022529007 A JP2022529007 A JP 2022529007A JP 2022529007 A JP2022529007 A JP 2022529007A JP 2023503046 A5 JP2023503046 A5 JP 2023503046A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- nucleic acid
- sequence
- aav vector
- acid sequence
- 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 (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962937583P | 2019-11-19 | 2019-11-19 | |
| US201962937556P | 2019-11-19 | 2019-11-19 | |
| US62/937,556 | 2019-11-19 | ||
| US62/937,583 | 2019-11-19 | ||
| US202063023570P | 2020-05-12 | 2020-05-12 | |
| US63/023,570 | 2020-05-12 | ||
| PCT/US2020/061223 WO2021102107A1 (en) | 2019-11-19 | 2020-11-19 | Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023503046A JP2023503046A (ja) | 2023-01-26 |
| JP2023503046A5 true JP2023503046A5 (enExample) | 2023-11-28 |
Family
ID=75981066
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022529007A Pending JP2023503046A (ja) | 2019-11-19 | 2020-11-19 | ポンペ病およびリソソーム障害を処置するための肝臓特異的プロモーターを含む治療用アデノ随伴ウイルス |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230038520A1 (enExample) |
| EP (1) | EP4061946A4 (enExample) |
| JP (1) | JP2023503046A (enExample) |
| KR (1) | KR20220098384A (enExample) |
| CN (1) | CN116096895A (enExample) |
| AU (1) | AU2020388634A1 (enExample) |
| CA (1) | CA3159018A1 (enExample) |
| IL (1) | IL293068A (enExample) |
| MX (1) | MX2022005916A (enExample) |
| PH (1) | PH12022551229A1 (enExample) |
| WO (1) | WO2021102107A1 (enExample) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2022513067A (ja) * | 2018-11-16 | 2022-02-07 | アスクレピオス バイオファーマシューティカル， インコーポレイテッド | ポンペ病を処置するための治療用アデノ随伴ウイルス |
| WO2023288301A1 (en) * | 2021-07-15 | 2023-01-19 | Cornell University | Engineered biomolecules for nutrient reprogramming |
| AR126877A1 (es) * | 2021-08-25 | 2023-11-22 | Canbridge Pharmaceuticals Inc | PARTÍCULAS DE AAV QUE COMPRENDEN UNA PROTEÍNA DE LA CÁPSIDE HEPATOTRÓPICA Y a-GLUCOSIDASA ÁCIDA (GAA) Y SU USO PARA TRATAR LA ENFERMEDAD DE POMPE |
| CA3229998A1 (en) * | 2021-08-25 | 2023-03-02 | Canbridge Pharmaceuticals, Inc. | Aav particles comprising a liver-tropic capsid protein and alpha-galactosidase and their use to treat fabry disease |
| WO2023139496A1 (en) * | 2022-01-21 | 2023-07-27 | Pfizer Inc. | Gene therapy for gaucher disease |
| CA3250992A1 (en) * | 2022-02-03 | 2023-08-10 | Astellas Gene Therapies, Inc. | COMPOSITIONS AND METHODS FOR AN IMPROVED TREATMENT OF POMPE DISEASE |
| CN119095612A (zh) * | 2022-02-25 | 2024-12-06 | 阿斯克肋匹奥生物制药公司 | 用于在长期停止gaa酶替代疗法的情况下治疗庞贝病的治疗性腺相关病毒 |
| WO2023172975A1 (en) * | 2022-03-08 | 2023-09-14 | University Of Massachusetts | Methods of raav packaging |
| US20250213726A1 (en) * | 2022-03-18 | 2025-07-03 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus using codon optimized nucleic acid encoding alpha-glucosidase (gaa) for treating pompe disease, with signal peptide modifications |
| WO2024055272A1 (zh) * | 2022-09-16 | 2024-03-21 | 复旦大学附属中山医院 | 能高效表达目的基因的mRNA载体系统、其构建及应用 |
| WO2024212961A1 (en) * | 2023-04-10 | 2024-10-17 | Skyline Therapeutics (Shanghai) Co., Ltd. | Recombinant aav for the gene therapy of pompe disease |
| CN121358865A (zh) * | 2023-04-20 | 2026-01-16 | 阿斯克生物公司 | 肝脏特异性调控核酸序列 |
| KR20240159745A (ko) | 2023-04-28 | 2024-11-06 | 아주대학교산학협력단 | 유산균 유래 nadh 산화효소를 이용한 비알코올성 지방간염의 유전자 치료제 |
| WO2025018822A1 (ko) * | 2023-07-20 | 2025-01-23 | 제닉스큐어 주식회사 | 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물 |
| KR102814185B1 (ko) * | 2023-07-20 | 2025-06-12 | 제닉스큐어 주식회사 | 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물 |
| IL326018A (en) * | 2023-07-28 | 2026-03-01 | Regeneron Pharma | Introduction of anti-TFR:GAA and anti-CD63:GAA for the treatment of Pompe disease |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2001266740A1 (en) * | 2000-06-06 | 2001-12-17 | Genelabs Technologies, Inc. | Promoters for regulated gene expression |
| ES2371913T3 (es) * | 2003-01-22 | 2012-01-11 | Duke University | Constructos mejorados para expresar polipéptidos lisosomales. |
| US7521211B2 (en) * | 2005-04-05 | 2009-04-21 | Regeneron Pharmaceuticals, Inc | IGF-1 and IGF-2 chimeric polypeptides and therapeutic uses thereof |
| EP2162540A2 (en) * | 2007-05-22 | 2010-03-17 | Amgen Inc. | Compositions and methods for producing bioactive fusion proteins |
| JP5627571B2 (ja) * | 2008-05-07 | 2014-11-19 | バイオマリン ファーマシューティカル インコーポレイテッド | リソソーム標的化ペプチドおよびその使用 |
| AU2010366066B2 (en) * | 2010-12-22 | 2016-01-14 | Fondazione Telethon | Therapeutic strategies to treat CNS pathology in mucopolysaccharidoses |
| MX2013012345A (es) * | 2011-04-22 | 2015-05-07 | Genzyme Corp | Alfa glucosidasa acida modificada con procesamiento acelerado. |
| KR102423442B1 (ko) * | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| EP3293260A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3293259A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| CN111936621B (zh) * | 2018-02-05 | 2025-05-13 | 奥登茨治疗公司 | 转录调控元件和它的用途 |
| US12258597B2 (en) * | 2018-02-07 | 2025-03-25 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for therapeutic protein delivery |
| BR112020021962A2 (pt) * | 2018-04-30 | 2021-01-26 | Amicus Therapeutics, Inc. | construtos para terapia gênica e métodos de uso |
| JP2022513067A (ja) * | 2018-11-16 | 2022-02-07 | アスクレピオス バイオファーマシューティカル， インコーポレイテッド | ポンペ病を処置するための治療用アデノ随伴ウイルス |
-
2020
- 2020-11-19 KR KR1020227020169A patent/KR20220098384A/ko active Pending
- 2020-11-19 JP JP2022529007A patent/JP2023503046A/ja active Pending
- 2020-11-19 PH PH1/2022/551229A patent/PH12022551229A1/en unknown
- 2020-11-19 CA CA3159018A patent/CA3159018A1/en active Pending
- 2020-11-19 EP EP20890917.6A patent/EP4061946A4/en active Pending
- 2020-11-19 AU AU2020388634A patent/AU2020388634A1/en active Pending
- 2020-11-19 WO PCT/US2020/061223 patent/WO2021102107A1/en not_active Ceased
- 2020-11-19 MX MX2022005916A patent/MX2022005916A/es unknown
- 2020-11-19 IL IL293068A patent/IL293068A/en unknown
- 2020-11-19 US US17/778,175 patent/US20230038520A1/en active Pending
- 2020-11-19 CN CN202080093548.9A patent/CN116096895A/zh active Pending
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