Experimental drug VX-864 aims to fix rare lung and liver disease
Disease control
Terminated
This study tested an experimental drug called VX-864 in 14 adults with Alpha-1 antitrypsin deficiency who have the PiZZ genetic type. The goal was to see if the drug could raise levels of a protective protein in the blood and reduce harmful protein clumps in the liver. The study …
Phase: PHASE2 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 15, 2026 11:56 UTC