Neurogene reports interim data in Pediatric Cohort from NGN-401 trial

Neurogene (NGNE) “announced positive, updated interim clinical data in the pediatric cohort (ages 4-10 cohort) from its ongoing Phase 1/2 trial evaluating NGN-401 gene therapy for the treatment of females with Rett syndrome.” “Updated interim data from our Phase 1/2 trial of NGN-401 show that all pediatric participants, regardless of baseline disease severity, have gained developmental milestones/skills or experienced functional gains, with durability and continued skill acquisition over time,” said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. “These skill gains far exceed the bar for our Embolden registrational trial, and coupled with NGN-401’s tolerability profile at the 1E15 vg dose, create the potential for a best-in-class gene therapy for Rett syndrome. It is gratifying to see the benefits of our strategy to use local delivery to the brain coupled with the naturally occurring full-length human gene as a key to unlocking meaningful efficacy in this devastating disorder. Moreover, we are the only company evaluating a gene therapy in children with Rett syndrome as young as three years old in a single trial, which provides us an efficient path to market and first-in-class potential.”