Hemophilia B gene therapy from Pfizer proves effective in late-stage research

The rare inherited blood disorder haemophilia B can be treated with experimental gene therapy, according to U.S. pharmaceutical company Pfizer Inc (PFE.N), which reported success in a late-stage study.

According to study data, a single dose of the therapy reduced the rate of bleeding in patients with moderately severe to severe forms of haemophilia B better than the current standard of care.

The condition impairs the body’s production of the blood-clotting protein factor IX.