Perlmutter Lab

Transforming the understanding of misfolded protein diseases, David Perlmutter’s laboratory investigates the pathobiology of α1-antitrypsin deficiency, a rare disorder that drives chronic liver failure and hepatocellular carcinoma. Through groundbreaking studies on protein clearance and liver pathology, the team has developed a pipeline of drugs capable of eliminating misfolded proteins and reversing disease in model systems, with one therapy advancing to phase II/III clinical trials. This innovative drug class holds promise not only for liver disorders but also for age-related neurodegenerative diseases such as Alzheimer’s.