Sangamo, Pfizer Update Phase 1/2 Results for Hemophilia A Gene Therapy

• 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 activity kinetics that appear consistent with the first two patients in this dose cohort at similar early time points
• SB-525 showed dose-dependent increases in FVIII activity levels across all dose cohorts evaluated
• FDA recently granted regenerative medicine advanced therapy (RMAT) designation for SB-525 gene therapy to treat severe hemophilia A

https://www.marketscreener.com/PFIZER-23365019/news/Pfizer-Sangamo-and-Pfizer-Announce-Updated-Phase-1-2-Results-for-SB-525-Investigational-Hemophilia-28859370/