Partner Case Study: Accelerating Gene Editing Manufacturing

By ElevateBio and Beam Therapeutics

Beam Therapeutics and ElevateBio joined forces in March 2022 to pioneer the manufacturing of BEAM-101, a cutting-edge base editing therapy designed to treat sickle cell disease. This innovative therapy aims to replicate naturally occurring genetic variants linked to hereditary persistence of fetal hemoglobin, offering hope for a transformative treatment for this challenging condition. To bring BEAM-101 from the lab to clinical trials, Beam leveraged ElevateBio’s deep expertise in cell and gene therapy manufacturing, combining stringent quality controls with rapid development capabilities.

Producing gene-edited cell therapies like BEAM-101 involves complex challenges beyond traditional cell therapy processes. Base editing must maintain high editing precision while preserving cell viability, requiring significantly adapted manufacturing methods. Beam faced the additional pressure of an accelerated timeline, driven by the urgent need to prepare for imminent clinical trials and ensure quality was never compromised.

According to Brian Riley, Beam’s chief manufacturing officer, “Base editing represents a new frontier in genetic medicine, and its manufacturing demands match that innovation.” The collaboration with ElevateBio enables Beam to tackle these technical and timeline challenges head-on, moving swiftly to deliver a promising therapy to patients awaiting new treatment options.