Dicerna™ to Begin Clinical Development for Liver Disease Treatment

• Company Submitted Clinical Trial Application and Plans to Initiate Multicenter Phase 1/2 Trial in Third Quarter of 2019 
• Alpha-1 Antitrypsin Deficiency-Associated Liver Disease Program Broadens Dicerna’s Commitment to Addressing Chronic Liver Diseases

Dicerna^TM Pharmaceuticals, Inc. (Nasdaq: DRNA) (the “Company” or “Dicerna”), a leading developer of ribonucleic acid interference (RNAi) therapies, today announced it submitted a Clinical Trial Authorization (CTA) application to the Swedish Medical Products Agency (MPA) last week to conduct a first-in-human Phase 1/2 study of DCR-A1AT, an investigational therapy from the Company’s GalXC™ technology platform, for the treatment of alpha-1 antitrypsin (A1AT) deficiency-associated liver disease. A1AT deficiency is a genetic disorder that can cause liver disease in children and adults, leading to complications such as weight loss, fatigue, jaundice and life-threatening conditions such as cirrhosis. Patients with A1AT deficiency are also at risk for developing hepatocellular carcinoma.

https://www.biospace.com/article/releases/dicerna-to-begin-clinical-development-of-dcr-a1at-for-treatment-of-patients-with-alpha-1-antitrypsin-deficiency-associated-liver-disease/