Neurogene says on track to share updated clinical efficacy and safety data

In addition to its update on registrational trial design for Embolden study, the company states: “Neurogene (NGNE) previously shared that five additional participants had been dosed in the Phase 1/2 portion of the NGN-401 clinical trial during the first half of 2025. A total of 10 participants have received the 1E15 vg dose. There has been no evidence of hemophagocytic lymphohistiocytosis / hyperinflammatory syndrome in any participant at the 1E15 vg dose level. Neurogene remains on track to share updated clinical efficacy and safety data in the second half of 2025. As of March 31, 2025, Neurogene had cash, cash equivalents, and investments of $292.6 million. The Company’s current budget reflects a reallocation of capital from pipeline development to support pivotal trial activities for NGN-401. Neurogene now expects its existing resources will be sufficient to fund operations into early 2028.”