Beam unveils phase 1/2 data of base-editing therapy in sickle cell disease

Beam Therapeutics shared new safety and efficacy data from its phase 1/2 trial of its investigational base-editing therapy, BEAM-101, in patients with sickle cell disease with severe vaso-occlusive crises at the American Society of Hematology Annual Meeting and Exposition in San Diego.

Beam had teased the presentation back in November, sharing initial results from the BEACON phase 1/2 study evaluating the safety and efficacy of BEAM-10, a single dose of autologous CD34+ base edited hematopoietic stem cells. The analysis included four patients, all of whom experienced rapid and robust fetal hemoglobin induction by month 1 (>60%) and corresponding sickle hemoglobin reduction (≤36%) in non-transfused blood, which was sustained over time.

The updated data now includes seven patients treated with BEAM-101. The therapy continued to demonstrate robust and durable increases in fetal hemoglobin and reductions in sickle hemoglobin, rapid neutrophil and platelet engraftment, and normalized or improved markers of hemolysis. No vaso-occlusive crises were reported post-engraftment.

To date, Beam has enrolled more than 35 patients in the BEACON trial, and of these, 11 patients have been dosed with BEAM-101.

In a separate presentation at ASH, Beam shared data from an additional study, demonstrating that its engineered stem cell antibody evasion (ESCAPE) approach, using CD117 monoclonal antibody conditioning, enabled engraftment of base-edited hematopoietic stem cells and induced robust, durable production of fetal hemoglobin in a non-human primate model.

Beam says it looks forward to continuing to rapidly advance both programs for patients with sickle cell disease.