Editas shows better gene editing using Cas9 alternative for sickle cell, thalassemia

Editas is using an alternative to Cas9 to develop a differentiated sickle cell and β thalassemia CRISPR gene therapy. Preclinical data presented Monday at ASH suggest that ex vivo gene editing could be more effective via Cas12a.

Editas Medicine Inc. (NASDAQ:EDIT) said the therapy, EDIT-301, is the first to use Cas12a. The autologous human hematopoietic stem cell (HSC) therapy, which is in IND-enabling studies for sickle cell disease and β thalassemia, is produced using an optimized Cas12a to disrupt the binding site for BCL11A within the HBG1 and HBG1 promoter. BCL11A is a transcription factor that prevents fetal hemoglobin expression...