Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in it’s entirety here..
Spark Therapeutics and Pfizer announced updated results from the Phase 1/2 study of SPK-9001, an investigational recombinant gene transfer product in hemophilia B, at the 58th American Society of Hematology (ASH) Annual Meeting, in San Diego.
Nine adult hemophilia B patients, aged 18 to 52 years, received a single dose of the gene therapy product engineered to enter their liver cells and stimulate production of factor IX. Patients participating in the current trial successfully maintained factor levels of about 30 percent.