Beam Therapeutics Sickle Cell Base Editing Works, Not Cause of Patient Death
Inside Precision Medicine December 10, 2024
At ASH 2024, the company unveiled Phase I/II safety and efficacy data for BEAM-101 and a preclinical update on their non-genotoxic approach (ESCAPE)
In a field bubbling with preclinical data and ideas on complex genome edits—including rewriting chunks of DNA—Beam Therapeutics has shown that just one nucleotide change can change an entire life.
At the 66th Annual Meeting and Exposition for the American Society of Hematology (ASH) in San Diego, California, Beam Therapeutics provided its first data on BEACON, the company’s clinical trial on sickle cell disease that uses base editing ex vivo on autologous hematopoietic stem cells (HSCs).
Results from the first seven patients show that BEAM-101 can significantly change the hemoglobin profile and provide rapid engraftment with a...