CRISPR-Based Therapy Receives Rare Pediatric Disease Designation for Sickle Cell Disease

The FDA granted Rare Pediatric Disease (RPD) designation to EDIT-301, an experimental, autologous cell therapy for the treatment of sickle cell disease (SCD). The RPD designation makes EDIT-301 eligible for an FDA priority review voucher if the agency approves the treatment’s biologics license application.

EDIT-301 is comprised of a patient’s CD34+ cells that have been genetically modified using a CRISPR/Cas12a ribonucleoprotein (RNP) to edit the HBG1/2 promoter region in the beta-globin locus. Red blood cells derived from EDIT-301–manufactured CD34+ cells have demonstrated a sustained increase in fetal hemoglobin production – also the goal of the accepted therapy, hydroxyurea – which may provide a durable benefit for individuals living with SCD.

Editas Medicine, the therapy’s manufacturer, plans to file an investigational new drug application for EDIT-301 by the end of 2020.

Source: Editas press release, August 24, 2020.