Beam Therapeutics Inc. reported data highlighting its optimized, closed and automated manufacturing process for its base-edited CD34+ hematopoietic stem and progenitor cell (HSPC) genetic medicines in a poster presentation at the European Hematology Association (EHA) Hybrid Congress. The optimized process is deployed for the manufacturing of BEAM-101 in the BEACON Phase 1/2 clinical trial in patients with severe sickle cell disease (SCD), and the data include both preclinical and GMP clinical manufacturing experience to date. Beam designed its automated CD34+ HSPC process for manufacturing of BEAM-101 to have the flexibility for a wide range of patient-starting material and variable cell numbers, while maintaining robust cell yield and high drug product quality suitable for use in the BEACON Phase 1/2 clinical trial.

Today's data highlight the following: The integration of key technologies has led to robust and improved process performance. Automation improved manufacturing execution, including increased cumulative yield and process consistency, and provided an up to three-fold increase in process capacity while reducing process duration, contamination risk and operator variability. Drug product reproducibly met the high viability threshold across 14 development runs using healthy and sickle cell trait donor cells and nine GMP clinical runs using SCD patient cells.

The use of base editing in an optimized, closed and automated process produced clinical drug product with consistently high CD34+ purity ranging from 84% to 95% and an editing rate ranging from 88% to 94%.