Eichler F, et al. - In this study, researchers report the initial outcomes of the STARBEAM study (ALD-102), an ongoing multicenter, single-group, open-label, phase 2–3 study of gene therapy with the Lenti-D drug product, which involves infusion of autologous CD34+ hematopoietic stem cells transduced ex vivo with the elivaldogene tavalentivec (Lenti-D) lentiviral vector that contains ABCD1 cDNA. Early outcomes of this investigation recommend that Lenti-D gene therapy may be a safe and effective option to allogeneic stem-cell transplantation in boys with early-stage cerebral adrenoleukodystrophy. Additional follow-up is needed to fully evaluate the duration of response and long-term safety.