CN119451694A - 编码组成型活性环状GMP-AMP合酶的mRNA及其脂质递送媒介物 - Google Patents

编码组成型活性环状GMP-AMP合酶的mRNA及其脂质递送媒介物 Download PDF

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Publication number
CN119451694A
CN119451694A CN202380050166.1A CN202380050166A CN119451694A CN 119451694 A CN119451694 A CN 119451694A CN 202380050166 A CN202380050166 A CN 202380050166A CN 119451694 A CN119451694 A CN 119451694A
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composition
mrna
lipid
lnp
cgas
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Chinese (zh)
Inventor
E·戈斯林
D·日瓦基
J·周
A·尼基福罗夫
D·森古普塔
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Kona Treatment Co
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Kona Treatment Co
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CN202380050166.1A 2022-05-13 2023-05-12 编码组成型活性环状GMP-AMP合酶的mRNA及其脂质递送媒介物 Pending CN119451694A (zh)

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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CN119451694A true CN119451694A (zh) 2025-02-14

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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)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121495900A (zh) * 2026-01-13 2026-02-10 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

Families Citing this family (1)

* 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

Family Cites Families (3)

* 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

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121495900A (zh) * 2026-01-13 2026-02-10 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
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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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
IL316957A (en) 2025-01-01
KR20250023373A (ko) 2025-02-18

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