DNS-6288

DNS-6288 is a small molecule ryanodine receptor 2 (RyR2) stabilizer under development by Agiana Pharmaceuticals for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT). CPVT is a rare, life-threatening genetic arrhythmia triggered by physical or emotional stress, caused by mutations that lead to leaky RyR2 calcium channels in the heart. DNS-6288 specifically targets and stabilizes the RyR2 channel in its closed state, preventing the spontaneous diastolic calcium release that initiates delayed afterdepolarizations (DADs) and subsequent ventricular arrhythmias. The drug has received Orphan Drug Designation from both the FDA and EMA, highlighting its potential as a targeted therapy for this orphan disease.