A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)

The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult subjects with severe sickle cell disease (SCD).The main goal of the study is to evaluate the efficacy of EDIT-301 by measuring the difference (pre-treatment versus post-treatment) in the annualized rates of severe vaso-occlusive events (VOEs) requiring medical attention. Safety and tolerability will also be assessed.

Study treatment consists of CD34+ mobilization, myeloablative conditioning, administration of EDIT-301 and 2 years of follow-up.

The study drug is EDIT-301, a novel, autologous, clustered regularly interspaced short palindromic repeats, gene-edited CD34+ HSPC (hereafter referred to as CD34+ cells) therapy product.