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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nucleic acid
gaa
sequence
polypeptide
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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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    • C12N9/2405—Glucanases
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    • C12N2750/14011—Parvoviridae
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    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • 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
US201962937556P 2019-11-19 2019-11-19
US201962937583P 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 (3)

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JP2023503046A true JP2023503046A (ja) 2023-01-26
JP2023503046A5 JP2023503046A5 (https=) 2023-11-28
JPWO2021102107A5 JPWO2021102107A5 (https=) 2023-11-28

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US (1) US20230038520A1 (https=)
EP (1) EP4061946A4 (https=)
JP (1) JP2023503046A (https=)
KR (1) KR20220098384A (https=)
CN (1) CN116096895A (https=)
AU (1) AU2020388634A1 (https=)
CA (1) CA3159018A1 (https=)
IL (1) IL293068A (https=)
MX (1) MX2022005916A (https=)
PH (1) PH12022551229A1 (https=)
WO (1) WO2021102107A1 (https=)

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WO2020102645A1 (en) * 2018-11-16 2020-05-22 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease
US20240287148A1 (en) * 2021-07-15 2024-08-29 Cornell University Engineered biomolecules for nutrient reprogramming
KR20240073025A (ko) * 2021-08-25 2024-05-24 캔브리지 파마슈티컬스, 인크. 간-향성 캡시드 단백질 및 알파-갈락토시다제를 포함하는 aav 입자 및 파브리 질환을 치료하기 위한 이의 용도
TW202338095A (zh) * 2021-08-25 2023-10-01 美商北海康成製藥有限公司 包含肝臟向性衣殼蛋白質及酸α－葡萄糖苷酶（ＧＡＡ）之ＡＡＶ顆粒及其用於治療龐貝氏病（ＰＯＭＰＥ ＤＩＳＥＡＳＥ）之用途
MX2024009003A (es) * 2022-01-21 2024-09-17 Astrazeneca Ireland Ltd Terapia genica para la enfermedad de gaucher.
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WO2023164060A2 (en) * 2022-02-25 2023-08-31 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease with long term cessation of gaa enzyme replacement therapy
WO2023172975A1 (en) * 2022-03-08 2023-09-14 University Of Massachusetts Methods of raav packaging
EP4493704A2 (en) * 2022-03-18 2025-01-22 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
EP4698655A1 (en) * 2023-04-20 2026-02-25 AskBio Inc. Liver-specific regulatory nucleic acid sequences
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
AU2024317483A1 (en) * 2023-07-28 2026-01-29 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease

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US20230038520A1 (en) 2023-02-09
IL293068A (en) 2022-07-01
KR20220098384A (ko) 2022-07-12
TW202132570A (zh) 2021-09-01
WO2021102107A1 (en) 2021-05-27
EP4061946A4 (en) 2024-03-06
CA3159018A1 (en) 2021-05-27
CN116096895A (zh) 2023-05-09
MX2022005916A (es) 2022-08-04
PH12022551229A1 (en) 2023-07-31
AU2020388634A1 (en) 2022-06-23
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