Clinical DevelopmentNeurogene's NGN-401 shows impressive and durable benefits at a low dose for treating Rett syndrome, with all patients showing improvement.
Financial PositionNeurogene's financial position is robust, bolstered by substantial private placement proceeds, significantly extending its cash runway for future developments.
Regulatory ProgressThe FDA allowed Neurogene to proceed with low-dose NGN-401, confirming a favorable safety profile with only mild adverse events observed.