Positive data could expand use of Novartis’ gene therapy for SMA

On 30 December 2024, Novartis reported that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) reached its primary endpoint in a Phase III study in paediatric patients aged two to 17 with type II spinal muscular atrophy (SMA). The positive data could expand the eligible patient population for gene transfer therapy (STEER).