UniQure's Gene Therapy Boosts Clotting in Two Hemophiliacs
- Factor IX levels rose to 4.5% and 5.5% of what is normal
- Gene therapy would compete with Pfizer, Biogen products
UniQure NV, the Dutch biotechnology company that developed the first gene therapy approved in Europe, said its experimental treatment for hemophilia B increased blood clotting ability in two patients. The shares rose as much as 21 percent in trading before U.S. markets opened.
The therapy, known as AMT-060, was designed to fix a genetic flaw caused by missing or defective factor IX, a protein that clots blood. The two adult patients received a low dose of the treatment. Factor IX rose to 5.5 percent and 4.5 percent of the normal level from less than 2 percent in the patients, the company said in a statement Thursday. Three other people who received a low dose of the therapy in the trial haven’t yet reached the full 12 weeks of follow-up, UniQure said.