Pfizer and Sangamo see hope in hemophilia data

An experimental gene therapy for hemophilia from Pfizer and Sangamo Therapeutics is showing promise: In a small, early trial, the one-time treatment boosted factor VIII, a protein that helps form blood clots and is deficient in people with hemophilia. Three patients who received the highest dosage saw their factor VIII levels return to normal, and their bleeds cut down to zero.

The trial was tiny — just 10 patients — but the magnitude of success suggests that the treatment could prove effective in later trials, and that the companies could join players like BioMarin and Spark Therapeutics in the race for curative options.