About Alpha-1 Antitrypsin Deficiency-Associated Liver Disease
Alpha-1 antitrypsin (AAT) deficiency is a rare, inherited disorder that can lead to liver disease in children and liver and lung disease in adults. In individuals with AAT deficiency-associated liver disease (AATLD), the condition can cause scarring of the liver, or cirrhosis, and may also result in the most common type of primary liver cancer known as hepatocellular carcinoma (HCC).
The SHINE Program
There are no available therapies specifically for AATLD, leaving patients limited to treatment options that cannot stop or reverse the resulting liver damage. Researchers are now turning their attention to this often unrecognized and underdiagnosed condition, and the Dicerna team is proud to be at the forefront of these efforts. SHINE, our clinical development program, currently includes a Phase 2 clinical study named ESTRELLA, which is evaluating an investigational RNAi therapy designed to address the underlying cause of AATLD.