Dicerna Announces Interim Results From Phase 1 Trial of Belcesiran for Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease
Data from this interim analysis showed dose-dependent reductions in serum alpha-1 antitrypsin (AAT) with administration of a single dose of belcesiran.
- Data from this interim analysis showed dose-dependent reductions in serum alpha-1 antitrypsin (AAT) with administration of a single dose of belcesiran.
- The primary treatment evaluation period for the final dose cohort (12.0 mg/kg) of belcesiran in the Phase 1 trial is ongoing.
- Alpha-1 antitrypsin (AAT) deficiency is a rare genetic condition caused by mutations in the SERPINA1 gene that results in disease of the liver and lungs.
- Belcesiran is a clinical-stage, subcutaneously administered, investigational GalXC RNAi therapy targeting alpha-1 antitrypsin (AAT) that is in development for the treatment of AAT deficiency-associated liver disease (AATLD).