Neurogene Stock Tanks After Adverse Event In Early-Stage Rett Syndrome Gene Therapy Trial

On Monday, Neurogene Inc. NGNE released interim clinical data in the first four participants in the low-dose cohort of its ongoing Phase 1/2 open-label trial of NGN-401 gene therapy for female pediatric patients with Rett syndrome, a rare genetic neurological and developmental disorder.Low-dose (1E15 vg) and high-dose (3E15 vg) NGN-401 have been well-tolerated with a favorable safety profile in the first seven pediatric participants (N=5 low-dose; N=2 high-dose):No treatment-related serious adverse events ( ...