FDA grants Rare Pediatric Disease status to CRISPR-based therapy EDIT-301 for Sickle Cell Disease

The FDA granted Rare Pediatric Disease (RPD) designation for EDIT-301 (Editas Medicine, Inc.), an experimental, autologous cell medicine intended for the treatment of sickle cell disease (SCD). EDIT-301 comprises sickle patient haematopoietic stem/progenitor cells (HSPCs) that are genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) to edit the γ-globin gene promoter region in the beta-globin locus. Red blood cells (RBCs) derived from EDIT-301 HSPCs demonstrate a sustained increase in foetal haemoglobin (HbF) production, which inhibits polymerisation of sickle haemoglobin (HbS) in RBCs, a hallmark of SCD, and has the potential to provide long-term treatment benefits for people living with SCD. The Company expects to file to the FDA for Investigational New Drug (IND) for SCD by the end of 2020. Source: ASH News