ALXN2350

**ALXN2350** is an investigational gene therapy developed by Alexion Pharmaceuticals (AstraZeneca Rare Disease) for BAG3 mutation-associated dilated cardiomyopathy (DCM), a genetic form of heart failure characterized by left ventricular enlargement and weakened pumping. It consists of an AAV9 viral vector delivering a functional BAG3 transgene to restore BAG3 protein expression, which is essential for cardiomyocyte protein quality control and autophagy; mutations in BAG3 (Bcl2-associated athanogene 3) disrupt these processes, leading to DCM progression. Administered as a single intravenous infusion, it is currently in a Phase 1/2 open-label trial (DCMRestore, NCT07218887) evaluating safety, tolerability, and efficacy in adults with pathogenic BAG3 mutations, with dose-finding and expansion cohorts.[1][2][5][9]

Other names

ALXN 2350ALXN2350ALXN-2350