JP2025510017A5 - - Google Patents

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Publication number
JP2025510017A5
JP2025510017A5 JP2024555349A JP2024555349A JP2025510017A5 JP 2025510017 A5 JP2025510017 A5 JP 2025510017A5 JP 2024555349 A JP2024555349 A JP 2024555349A JP 2024555349 A JP2024555349 A JP 2024555349A JP 2025510017 A5 JP2025510017 A5 JP 2025510017A5
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JP
Japan
Application number
JP2024555349A
Other languages
Japanese (ja)
Other versions
JP2025510017A (ja
JPWO2023177885A5 (https=
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2023/015531 external-priority patent/WO2023177885A2/en
Publication of JP2025510017A publication Critical patent/JP2025510017A/ja
Publication of JP2025510017A5 publication Critical patent/JP2025510017A5/ja
Publication of JPWO2023177885A5 publication Critical patent/JPWO2023177885A5/ja
Pending legal-status Critical Current

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JP2024555349A 2022-03-18 2023-03-17 シグナルペプチド改変を有する、ポンペ病を処置するためのアルファ-グルコシダーゼ(gaa)をコードするコドン最適化核酸を使用する治療用アデノ随伴ウイルス Pending JP2025510017A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202263321336P 2022-03-18 2022-03-18
US63/321,336 2022-03-18
US202263348862P 2022-06-03 2022-06-03
US63/348,862 2022-06-03
US202363444804P 2023-02-10 2023-02-10
US63/444,804 2023-02-10
PCT/US2023/015531 WO2023177885A2 (en) 2022-03-18 2023-03-17 Therapeutic adeno-associated virus using codon optimized nucleic acid encoding alpha-glucosidase (gaa) for treating pompe disease, with signal peptide modifications

Publications (3)

Publication Number Publication Date
JP2025510017A JP2025510017A (ja) 2025-04-14
JP2025510017A5 true JP2025510017A5 (https=) 2026-03-26
JPWO2023177885A5 JPWO2023177885A5 (https=) 2026-03-26

Family

ID=88024211

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024555349A Pending JP2025510017A (ja) 2022-03-18 2023-03-17 シグナルペプチド改変を有する、ポンペ病を処置するためのアルファ-グルコシダーゼ(gaa)をコードするコドン最適化核酸を使用する治療用アデノ随伴ウイルス

Country Status (11)

Country Link
US (1) US20250213726A1 (https=)
EP (1) EP4493704A2 (https=)
JP (1) JP2025510017A (https=)
KR (1) KR20250005569A (https=)
CN (1) CN119213137A (https=)
AU (1) AU2023235203A1 (https=)
CA (1) CA3254578A1 (https=)
IL (1) IL315711A (https=)
MX (1) MX2024011396A (https=)
TW (1) TW202403048A (https=)
WO (1) WO2023177885A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE521701T1 (de) * 2003-01-22 2011-09-15 Univ Duke Verbesserte konstrukte zur expression lysosomaler polypeptide
EP3293259A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
WO2020102667A2 (en) * 2018-11-16 2020-05-22 Asklepios Biopharmaceutical, Inc. Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence
CA3159018A1 (en) * 2019-11-19 2021-05-27 Michael W. O'CALLAGHAN Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders

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