Etedesiran is a component of the AOC drug Delpacibart etedesiran, formed by the reaction of an siRNA that induces cleavage of mRNA encoding myotonic dystrophy protein kinase (MTPK or DMPK ) with SMCC linker. Etedesiran carries a maleimide group at its terminus, which can react with cysteine or lysine and is used for the synthesis of AOC drugs. Etedesiran is applicable to research related to myotonic dystrophy type 1 [1].
In Vivo, Etedesiran can be used for synthesizing Delpacibart etedesiran, which dose-dependently and significantly reduces total DMPK mRNA levels in multiple skeletal muscles, hearts, and gastrointestinal tracts of untreated cynomolgus monkeys[1].
References:
[1]. Kwan TT, et al. Delpacibart etedesiran improves the molecular pathology of myotonic dystrophy type 1 in the phase 1/2 MARINA study. Mol Ther. Published online March 11, 2026.
In Vivo, Etedesiran can be used for synthesizing Delpacibart etedesiran, which dose-dependently and significantly reduces total DMPK mRNA levels in multiple skeletal muscles, hearts, and gastrointestinal tracts of untreated cynomolgus monkeys[1].
References:
[1]. Kwan TT, et al. Delpacibart etedesiran improves the molecular pathology of myotonic dystrophy type 1 in the phase 1/2 MARINA study. Mol Ther. Published online March 11, 2026.