Baxter: ISTH Congress (Baxter) - Jul 3, 2015 - "The ongoing BAX 335 Phase 1/2 study has escalated to 3rd dose cohort. An additional 5 patients will be dosed"; "Sustained expression of 20-25% for 1 year was observed in cohort 2"; "Peak FIX activity level >50% achieved in Cohort 3 but persistent, stable expression was not achieved in the first two patients in this cohort"; "Dose-dependent immune responses were observed in two patients in cohort 3 and treated with corticosteroid. FIX expression has subsequently declined in both subjects, likely due to delayed start of corticosteroid"; "No inhibitors observed in any of the subjects treated to date"; "Conclusion: Hemostatically effective plasma FIX activity levels were achieved in cohort 2 and 3 with sustained high level expression in one patient (20-25%)"
P1/2 data • Hemophilia
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