Clinical Trial ProgressNeurogene announced pivotal trial design appears to offer a clear path to show a benefit for Rett patients treated with NGN-401.
Financial PlanningCash reserves at the end of the period were $274.5M, expected to provide financial runway into early 2028, indicating strong financial planning.
Safety ProfileNo cases of hemophagocytic lymphohistiocytosis (HLH) or hyperinflammatory syndrome have been observed in the 10 patients treated at the 1E15 vg dose level, indicating a favorable safety profile.