Spark’s gene therapy data answer some burning questions — and raise a few more
Patients with hemophilia rely on regular injections of synthetic proteins to boost clotting. A gene therapy, if successful, would upend treatment.
An investigational hemophilia A treatment from Spark Therapeutics reduced bleeds by nearly 100 percent in a small clinical trial, improving upon earlier data that left investors unsatisfied. But it still remains to be seen whether Spark’s gene therapy can beat out a rival effort underway at BioMarin.
In a study on 12 patients, Spark’s one-time treatment boosted the production of factor VIII, a protein
