This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant (HSCT) in subjects with severe SCD. Planned study subjects will be comprised of male and female adult subjects with severe SCD, from 18 to 50 years of age, inclusive.
The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult participants with severe sickle cell disease (SCD).