Promising results on a phase 2 clinical study entitled “Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency” was recently published in The New England Journal of Medicine.
Alpha1-antitrypsin deficiency is an inherited, proteotoxic disorder caused by accumulation of mutated alpha1-antitrypsin protein (AAT). In this phase 2, open label clinical study, Strnad et al. demonstrated that fazirsiran, a hepatocyte-targeted small interfering RNA, decreases AAT production as well as hepatic AAT accumulation. The treatment was well tolerated and led to improvement in several liver injury markers.
Congratulations to Pavel Strnad (project A03) and Co-Author Christian Trautwein (projects A06, A08) on the successful publication of this study in this highly respected journal!