Beam Therapeutics unveiled data studying its base editing technology across two approaches to treating sickle cell disease, highlighting the potential of the technology to enable new therapeutic possibilities for those suffering from serious diseases.
The data shared in the press release includes initial results from the BEACON phase 1/2 study evaluating the safety and efficacy of a single dose of autologous CD34+ base edited hematopoietic stem cells (BEAM-101) in patients with sickle cell disease with severe vaso-occlusive crises. Six patients were dosed and 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.
One patient died four months after receiving the BEAM-101 infusion due to respiratory failure that Beam determined to be likely related to busulfan conditioning and deemed unrelated to BEAM-101. Pulmonary complications are a known side effect of chemotherapy, such as busulfan, in patients undergoing stem cell transplantation.
Beam also shared data from an additional study investigating whether its engineered stem cell antibody evasion (ESCAPE) approach, using CD117 monoclonal antibody conditioning, could successfully achieve long-term engraftment and induce robust levels of HbF in an immunocompetent host. Researchers used a non-human primate model where autologous CD34+ hematopoietic stem and progenitor cells were multiplex edited to target a single epitope on CD117 and the promoter regions of HBG1/2, allowing cells to avoid detection by the conditioning mAb while upregulating HbF. The data suggest that ESCAPE, combined with CD117 mAb conditioning and selection, can achieve long-term engraftment and induce high levels of HbF, offering a promising alternative to traditional genotoxic conditioning in autologous HSCT.
The full data, including the first clinical data from a Beam program, will be shared in the form of multiple oral and poster presentations at the 66th American Society of Hematology Annual Meeting and Exposition, taking place December 7-10 in San Diego.