Novartis has revealed new interim data from its Phase 3 trial investigating the efficacy of its gene therapy Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) in the treatment of spinal muscular atrophy (SMA) Type 1 in patients under six months old.
The data revealed that, following a one-time intravenous infusion of the therapy, Zolgensma “successfully transduced intended targets in the central nervous system (CNS) and provided widespread SMN expression comparable to tissue from unaffected individual.”
