A Game-Changing Breakthrough Could Be Coming for Hemophilia Patients
Patients diagnosed with hemophilia B suffer a lifetime of treatment, but that could change based on recently reported data from an early stage trial Pfizer (NYSE: PFE) and Spark Therapeutics (NASDAQ: ONCE) are conducting. The two companies are teamed up on SPK-9001, a gene therapy that hopes to deliver a "one-and-done" functional cure for the disease. This week, The New England Journal of Medicine, a prestigious industry journal, published updated results from a 10-patient study that were pretty remarkable. Can hemophilia become a curable disease?
The results from the company's phase 1/2 study show that a single dose of SPK-9001 had patients producing their own clotting factor, and as a result, the number of bleeds per year for these patients fell by 97% and the need for an infusing clotting factor dropped by 99%. Previously, these patients were experiencing an average 11.1 bleeding events per year, but after receiving SPK-9001, they suffered an average of just 0.4 per year.
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Source: Fool.com
