JP2023503046A - 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 - Google Patents

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 Download PDF

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JP2023503046A
JP2023503046A JP2022529007A JP2022529007A JP2023503046A JP 2023503046 A JP2023503046 A JP 2023503046A JP 2022529007 A JP2022529007 A JP 2022529007A JP 2022529007 A JP2022529007 A JP 2022529007A JP 2023503046 A JP2023503046 A JP 2023503046A
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マイケル ロバーツ，
フアン マヌエル イグレシアス，
アンナ トレチャコヴァ，
マイケル ダブリュー． オカラガン，
アキッレ フランソワ，
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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
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    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
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    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)

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JP2022529007A 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 Pending JP2023503046A (ja)

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

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JP2023503046A true JP2023503046A (ja) 2023-01-26
JP2023503046A5 JP2023503046A5 (enExample) 2023-11-28

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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)

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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
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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

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MX2022005916A (es) 2022-08-04
CN116096895A (zh) 2023-05-09
EP4061946A1 (en) 2022-09-28
EP4061946A4 (en) 2024-03-06
PH12022551229A1 (en) 2023-07-31
TW202132570A (zh) 2021-09-01
KR20220098384A (ko) 2022-07-12
IL293068A (en) 2022-07-01
CA3159018A1 (en) 2021-05-27
AU2020388634A1 (en) 2022-06-23
WO2021102107A1 (en) 2021-05-27

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