Biotech firms rush to develop gene-editing cure for rare alpha-1 antitrypsin deficiency

A wave of biotech companies is intensifying efforts to cure alpha-1 antitrypsin deficiency (AATD), a rare liver and lung disorder, using gene-editing approaches.

Companies such as Beam, Wave Life Sciences, Korro Bio, Tessera and Yoltech are each targeting the single DNA mutation that causes the disease, hoping to correct the faulty gene.

Analysts estimate the market could reach several billion dollars, and firms are committing billions of dollars to research and development in the hope of capturing that value.

The race is complicated by patent disputes, startup defections, arbitration and growing competition between U.S. and Chinese drug makers.