STAT Plus: Experimental gene therapy for hemophilia, developed by Pfizer and Sangamo, shows early promise
A one-time hemophilia A treatment from Pfizer (PFE) and Sangamo Therapeutics (SGMO) showed promising results in a small, early-stage study, pointing to a possible future in which patients might have a handful of curative options for the rare bleeding disorder.
In an ongoing 10-patient trial, the treatment boosted the production of factor VIII, a protein that is key to forming blood clots and is lacking in people with hemophilia A, according to data presented Saturday. Three patients getting the highest dose — the one Pfizer and Sangamo plan to use in future trials — saw their factor VIII levels returned to normal, and bleeds were reduced to zero. A fourth patient was dosed too recently to draw any conclusions.
Continue to STAT Plus to read the full story…
