The following is an excerpt from a press release from Spark Therapeutics. Read the press release in its entirety here.
Investigational SPK-8011 moving to Phase 3
As of the July 13 data cutoff, 12 participants in the Phase 1/2 trial have received a single administration of investigational SPK-8011, including two at a dose of 5×1011 vector genomes (vg)/kg body weight, three at a dose of 1×1012 vg/kg and seven at a dose of 2×1012 vg/kg. Across all participants, at all three doses, beginning four weeks after vector infusion, there has been a 97-percent reduction in annualized bleeding rate (ABR) and a 97-percent reduction in annualized infusion rate (AIR).
The first two trial participants, who have been followed for greater than one year, have shown stable FVIII activity levels since reaching a plateau for up to 66 weeks, with follow up ongoing. Additionally, there is evidence of a dose-dependent increase in mean FVIII activity levels across the three dose cohorts.
Read the press release in its entirety here.