Cash, cash equivalents and short-term investments as of September 30, 2025 were $265.4 million. We expect cash, cash equivalents and short-term investments to fund planned operations through the first quarter of 2028. “We significantly advanced our NGN-401 gene therapy clinical program for Rett syndrome over the past quarter, culminating in the recent dosing of the first participant in the Embolden registrational trial after initiating 12 of 13 sites that enable our plans to rapidly enroll the trial in the next three to six months,” stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene (NGNE). “The positive interim clinical data from the pediatric cohort of our NGN-401 Phase 1/2 trial shared yesterday, which showed multidomain, durable gains with continued developmental/milestone skill acquisition, underscore NGN-401’s therapeutic promise. With cash runway through the first quarter of 2028, we are in a strong financial position to bring a much-needed gene therapy to people with Rett syndrome.”
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