As BioMarin’s lead rival wanes, Pfizer and Sangamo post a promising update for hemophilia A gene therapy
Pfizer and its hemophilia A gene therapy partners at Sangamo have posted the latest update on their Phase I/II trial for SB-525 — and it continues to look good as their rivals at BioMarin cruise to a likely approval with a not-so-perfect leader.
Median Factor VIII expression levels for their gene therapy are running at 64.2% for the 5 patients now taking the 3e13 vg/kg dose. That includes patients up to 61 weeks after dosing, putting the crew on the trial at potentially better than par with BioMarin’s valrox at a year in, which has attracted plenty of questions as FVIII levels declined over 4 years to a median of 16.4%, as we reported yesterday.
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