ALXN-1540

**ALXN-1540** is an investigational mRNA-based therapy developed by Alexion (AstraZeneca Rare Disease) in collaboration with Moderna for Crigler-Najjar syndrome type 1 (CN-1), a rare genetic disorder caused by UGT1A1 deficiency leading to unconjugated hyperbilirubinemia. Delivered via lipid nanoparticles for hepatic expression, it encodes human UGT1A1 (hUGT1A1) to restore bilirubin glucuronidation and clearance, addressing the root cause rather than just managing symptoms like phototherapy. Early development faced safety challenges with lipid nanoparticle formulations, delaying human trials, and preclinical QSP modeling projected efficacy at doses like 0.5 mg/kg Q4W for sustained bilirubin reduction >5 mg/dL in CN-1 patients.[1][4][5][8][9]

Other names

WTX101WTX-101WTX 101