Early sickle cell data show promise, as Editas refines its pipeline

Data for EDIT-301 in sickle cell disease Tuesday marked the first clinical data using Editas’ gene editing nuclease AsCas12a. The company now faces the challenge of differentiating its asset from several more advanced programs.

Editas Medicine Inc. (NASDAQ:EDIT) said Tuesday the first sickle cell patient in the open-label Phase I/II RUBY trial showed fetal hemoglobin (or HbF) of 45.4% at five months and has not experienced any vaso-occlusive events since the treatment with EDIT-301 — clear signs of efficacy showing that EDIT-301 is working in autologous hematopoietic stem cell transplant...