Addressing A1AT deficiency-associated liver diseaseWe are developing DCR-A1AT, an investigational therapy from our GalXCTM technology platform, for the treatment of liver disease in patients with alpha-1 antitrypsin (A1AT) deficiency. A1AT deficiency is a genetic disorder that can cause liver disease in children and adults, leading to complications such as fatigue, jaundice or unintended weight loss. Patients with A1AT deficiency are also at risk of serious or life-threatening conditions such as cirrhosis, liver failure and hepatocellular carcinoma, and may develop lung disease, including chronic obstructive pulmonary disease (COPD) and emphysema. There are currently no approved therapies specifically designed to treat the liver manifestations of this condition.
- We are investigating the efficacy and safety of DCR-A1AT in a clinical trial development program, called SHINE. We are conducting a first-in-human Phase 1/2 study of DCR-A1AT, called ESTRELLA.
- ESTRELLA will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of DCR-A1AT in adult healthy volunteers and patients with A1AT deficiency-associated liver disease.
In June 2019, the Company submitted a clinical trial application to the Swedish Medical Products Agency for DCR-A1AT for the treatment of patients with A1AT deficiency-associated liver disease. We began enrolling healthy volunteers in ESTRELLA in the fourth quarter of 2019.
- 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).