Arrowhead Pharmaceuticals (ARWR) announced an exclusive worldwide license agreement with Madrigal Pharmaceuticals (MDGL) for ARO-PNPLA3, Arrowhead’s clinical stage RNA interference therapeutic designed to reduce liver expression of patatin-like phospholipase domain containing 3 as a potential treatment for patients with metabolic dysfunction-associated steatohepatitis. PNPLA3 has strong genetic and preclinical validation as a driver of fat accumulation and damage in the livers of patients who carry the I148M mutation. The I148M genetic variant is involved with the underlying pathophysiology and is a known risk factor for hepatic steatosis, steatohepatitis, elevated plasma liver enzyme levels, hepatic fibrosis, and cirrhosis.
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