The first two patients treated at the 3e13 vg/kg dose level rapidly achieved normal, sustained Factor VIII (FVIII) levels with no reported bleeding events and no factor usage for as long as 24 weeks of follow-up The two patients more recently treated at the 3e13 vg/kg dose level demonstrated FVIII
– The first two patients treated with the 3e13 vg/kg dose achieved stable Factor VIII (FVIII) levels demonstrating durability in the normal range through 44 and 37 weeks, respectively – All five patients in the 3e13 vg/kg dose cohort achieved normal range FVIII levels within 5-7 weeks following
– No adverse events related to investigational SAR445136 were reported – All four treated patients experienced increases in total hemoglobin, fetal hemoglobin and percent F cells; none required blood transfusions post engraftment BRISBANE, Calif. –(BUSINESS WIRE)–Dec.
– Isaralgagene civaparvovec, or ST-920, continued to be generally well tolerated across three dose cohorts in the five treated patients – In the first two dose cohorts, all four patients exhibited above normal α-Gal A activity, ranging from 3-fold to 15-fold above mean normal; patients in the first
– Isaralgagene civaparvovec, or ST-920, continued to be generally well tolerated across three dose cohorts in the six treated patients – The five longest treated patients exhibited elevated α-Gal A activity, ranging from nearly 3-fold to nearly 17-fold above mean normal, up to 15 months as at the
– Isaralgagene civaparvovec, or ST-920, continued to be generally well tolerated across four dose cohorts in the nine treated patients in the dose escalation phase. – All nine patients exhibited elevated α-Gal A activity, ranging from nearly 2-fold to 30-fold of mean normal, for up to 23 months