Drugs and FDA
Beam says base-edited sickle cell therapy met goal in trial
Beam said risto-cel met its primary goal of freedom from severe VOCs in data from 31 patients who completed treatment and 12 months of follow-up.
Beam Therapeutics said its base-edited sickle cell therapy, risto-cel, met its primary goal in a clinical trial. Beam said the primary goal was freedom from severe vaso-occlusive crises, or VOCs. The company also reported meeting secondary objectives, including reducing hospital stays associated with VOCs.
The results released Tuesday covered 31 patients who had completed treatment and 12 months of follow-up as of Oct. 20. Beam did not release specific details of the results, BioPharma Dive reported. The outlet said the findings could position Beam to submit an FDA application as soon as the end of the year.
