Addressing AAT deficiency-associated liver disease (AATLD)
Belcesiran, is our GalXC™ product candidate for the treatment of alpha-1 antitrypsin (AAT) deficiency-associated liver disease (AATLD). Alpha-1 antitrypsin deficiency (AATD) is a rare, inherited disorder that can lead to liver disease in children and liver and lung disease in adults. The condition is caused by mutations in the SERPINA1 gene. In people with AATLD, the liver produces an abnormal version of the AAT protein, which is prone to aggregation in the liver. This accumulation of mutated AAT in the liver can lead to liver disease. Individuals with AATLD also have an increased risk of having lung disease. There are currently no approved therapies to treat AATLD. We are currently evaluating Belcesiran in a Phase 1 clinical study and initiated a Phase 2 clinical trial of Belcesiran in the first half of 2021.
Belcesiran SHINE clinical development program
We are investigating belcesiran (DCR-A1AT) as part of a clinical development program called SHINE.
- We are investigating the efficacy and safety of belcesiran (DCR-A1AT) in a Phase 1 study under the SHINE clinical trial development program.
- SHINE includes a Phase 2 clinical trial, called ESTRELLA, which we initiated in the first half of 2021 to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of belcesiran in patients with AAT deficiency-associated liver disease.
- Sandhaus RA, Turino G, Brantly ML, et al. The Diagnosis and Management of Alpha-1 Antitrypsin Deficiency in the Adult. Journal of the COPD Foundation 2016; 3(3).