Abstract
This is a multi-site, observational study to evaluate the long-term safety and efficacy of CTX001 in subjects who received CTX001 in Study CTX001-111 (NCT03655678) or VX21-CTX001-141 (transfusion-dependent β-thalassemia [TDT] studies) or Study CTX001-121 (NCT03745287) or VX21-CTX001-151 (severe sickle cell disease [SCD] studies; NCT05329649).
