What AI says about Skysona

SKYSONA is a one-time gene therapy for boys aged 4–17 with early, active cerebral adrenoleukodystrophy (CALD) who lack a matched donor for allogeneic hematopoietic stem cell transplant. It uses the patient's own blood stem cells to add functional copies of the ABCD1 gene, enabling production of ALDP to break down very long-chain fatty acids and slow neurologic decline.