Neurogene Adjusts Rett Syndrome Gene Therapy Trials After High-Dose Setback

Neurogene faced a tragic setback in its high-dose gene therapy trial for Rett syndrome after a patient succumbed to complications following the administration of their NGN-401 therapy[1]. This patient, the third to receive a high dose, developed a rare hyperinflammatory syndrome associated with adeno-associated virus (AAV) vectors, which are common in gene therapies[1][2]. In response, Neurogene has halted enrollments for the high-dose cohort, opting instead to focus on the safety and promising results observed in low-dose trials[2]. The FDA has allowed trials to continue using the lower dose, which has shown potential for a safe and effective treatment pathway[1].