Posted by Michael Wonder on 30 Apr 2026
Expression Therapeutics receives FDA fast track and rare paediatric disease designations for investigational stem cell therapy for haemophilia A
29 April 2026 - Expression Therapeutics today announced that the US FDA has granted fast track designation and rare paediatric disease designation to its investigational therapy.
Dual designations affirm significant unmet need; multi year Phase 1 data published in the New England Journal of Medicine underpin advancement to Phase 2.
