VX-264

VX-264 is an investigational cell therapy developed by Vertex Pharmaceuticals for the treatment of type 1 diabetes. The therapy consists of fully differentiated, allogeneic human stem cell-derived pancreatic islet cells encapsulated within a proprietary immunoprotective device. This device is designed to shield the implanted insulin-producing cells from both autoimmune attack and immune rejection, allowing them to restore endogenous insulin production without the need for systemic immunosuppression. In clinical trials, VX-264 was found to be generally safe and well tolerated but did not achieve sufficient increases in C-peptide (a marker of insulin production) to deliver clinical benefit. As a result, development has been discontinued following Phase 1/2 studies[1][5][6].