Neurogene Halts High Dose NGN-401 Trials After Patient Death, Focuses on Low Dose Efficacy

A patient involved in the Neurogene clinical trial for Rett syndrome has died after receiving a high dose of their gene therapy candidate, NGN-401. The patient, who was the third to be administered this high dose, succumbed to a rare hyperinflammatory syndrome associated with high doses of adeno-associated virus (AAV), commonly used in such therapies[1]. Following this tragic event, Neurogene halted the high-dose trials but has been allowed by the FDA to continue testing with lower doses, which have demonstrated encouraging safety and efficacy results[2]. Chief Financial Officer Christine Mikail noted that the tripled dosage was part of an exploration into its effects, and some adverse reaction was anticipated[1].