Patient dies in Neurogene trial

A patient in Neurogene’s (Nasdaq: NGNE) ongoing Phase I/II trial for Rett syndrome - who was dosed on November 5 with 3E15 vg of NGN-401 - has now died from a treatment-related serious adverse event (SAE).

The woman was previously reported to be in critical condition following complications from a rare and life-threatening hyperinflammatory syndrome associated with systemic exposure to high doses of adeno-associated virus (AAV).

In a commitment to full transparency with the US Food and Drug Administration (FDA), Neurogene has proactively engaged with the agency following the company’s notification of the SAE.