KR20250023373A - 구성적으로 활성인 시클릭 GMP-AMP 신타제를 코딩하는 mRNA 및 이를 위한 지질 전달 비히클 - Google Patents

구성적으로 활성인 시클릭 GMP-AMP 신타제를 코딩하는 mRNA 및 이를 위한 지질 전달 비히클 Download PDF

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KR20250023373A
KR20250023373A KR1020247041462A KR20247041462A KR20250023373A KR 20250023373 A KR20250023373 A KR 20250023373A KR 1020247041462 A KR1020247041462 A KR 1020247041462A KR 20247041462 A KR20247041462 A KR 20247041462A KR 20250023373 A KR20250023373 A KR 20250023373A
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lipid
mrna
composition
antigen
glycero
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Korean (ko)
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에밀리 고셀린
다니아 지바키
조나단 차우
아나스타샤 니키포로브
데브루프 생업타
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코너 테라퓨틱스, 인크.
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KR1020247041462A 2022-05-13 2023-05-12 구성적으로 활성인 시클릭 GMP-AMP 신타제를 코딩하는 mRNA 및 이를 위한 지질 전달 비히클 Pending KR20250023373A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263341987P 2022-05-13 2022-05-13
US63/341,987 2022-05-13
PCT/US2023/066974 WO2023220746A1 (en) 2022-05-13 2023-05-12 Mrna encoding a constitutively-active cyclic gmp-amp synthase and lipid delivery vehicles for same

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KR20250023373A true KR20250023373A (ko) 2025-02-18

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US (1) US20250255988A1 (enExample)
EP (1) EP4522205A1 (enExample)
JP (1) JP2025516637A (enExample)
KR (1) KR20250023373A (enExample)
CN (1) CN119451694A (enExample)
AU (1) AU2023267834A1 (enExample)
CA (1) CA3256940A1 (enExample)
IL (1) IL316957A (enExample)
TW (1) TW202402303A (enExample)
WO (1) WO2023220746A1 (enExample)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025253381A1 (en) * 2024-06-04 2025-12-11 Edity Therapeutics Ltd. Cyclic gmp-amp synthase variants and use thereof
CN121495900B (zh) * 2026-01-13 2026-04-24 泰兴合全药业有限公司 一种环状gmp-amp合成酶突变体及其用于制备2′,3′-环状单磷酸鸟苷-单磷酸腺苷的方法

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3173092B1 (en) * 2015-04-22 2019-06-26 CureVac AG Rna containing composition for treatment of tumor diseases
EP3814487A4 (en) * 2018-06-29 2022-04-20 President and Fellows of Harvard College STRUCTURE OF THE HUMAN CGAS-DNA COMPLEX AND ITS USES
IL298765A (en) * 2020-06-11 2023-02-01 Etherna Immunotherapies Nv Lipid nanoparticles

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AU2023267834A1 (en) 2025-01-02
JP2025516637A (ja) 2025-05-30
CA3256940A1 (en) 2023-11-16
WO2023220746A1 (en) 2023-11-16
EP4522205A1 (en) 2025-03-19
TW202402303A (zh) 2024-01-16
US20250255988A1 (en) 2025-08-14
CN119451694A (zh) 2025-02-14
IL316957A (en) 2025-01-01

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