Edit-301: An Experimental Autologous Cell Therapy Comprising Cas12a-Rnp Modified Mpb-Cd34+Cells For The Potential Treatment Of Scd

Sickle cell disease (SCD) is an inherited blood disorder affecting approximately 100,000 individuals in the United States. Fetal hemoglobin (HbF) is a major modifier of SCD severity. Studies showed that individuals with compound heterozygosity for sickle hemoglobin (HbS) and hereditary persistence of fetal hemoglobin (HPFH) that expressed approximately 30% HbF did not show features of SCD. Therefore, we are developing EDIT-301, an experimental autologous cell therapy comprising CD34+ cells genetically modified using a Cas12a RNP (ribonucleoprotein) to promote HbF expression to treat SCD.