Neurogene reports 47 developmental milestone gains

• Neurogene (NASDAQ:NGNE) reported 47 total developmental milestone gains in its NGN-401 Rett syndrome gene therapy trial.
• All 10 participants showed clinical improvement, with no reported loss of milestones through up to 30 months of follow-up.
• The company said safety data remained generally consistent with known AAV gene therapy risks.

Neurogene (NASDAQ:NGNE) reported updated Phase 1/2 clinical data showing 47 total developmental milestone gains across 10 participants treated with NGN-401 gene therapy for Rett syndrome.

The update extends earlier trial results and adds longer follow-up data showing continued improvements across multiple functional domains without observed reversal of gains.

The dataset included 10 participants with 47 total milestone gains, averaging 4.7 per participant, with all participants gaining at least one milestone over the past 12 months and no loss of milestones reported through up to 30 months of follow-up.

The company also reported 100% improvement on the Clinical Global Impression-Improvement scale, along with statistically significant improvements in validated Rett syndrome functional measures.

Neurogene said NGN-401 remained generally well-tolerated at the 1E15 vg dose, with treatment-related adverse events mostly mild or moderate and no new serious adverse events reported since the prior data cutoff.