A decade ago, Pfizer began investing heavily in gene therapy, bringing in experimental treatments for a range of genetic diseases, pumping $800 million into “state-of-the-art” manufacturing facilities and announcing its intention to become an “industry leader” that would deliver “one-time, transformative therapies” for rare diseases.
Today, it’s not clear how much the company has to show for it.
On Wednesday, Pfizer announced data from its hemophilia A gene therapy. The results set up a potential approval, but questions about durability may deter most patients from getting it. A potentially curative hemophilia B gene therapy was approved in April but faces competition. A Duchenne muscular dystrophy treatment, the linchpin of the portfolio, failed to show any benefit in a large trial last month.
