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Neurogene issued a press release on November 11, 2024, reporting “interim clinical data in the first four participants in the low-dose cohort of its ongoing Phase 1/2 open-label trial designed to evaluate NGN-401 gene therapy for the treatment of female pediatric patients with Rett syndrome.” Although Neurogene characterized the data as “positive”, one of two patients given a high dose reported an emerging treatment-related serious adverse event that the Company described as “consistent with known risks of [adeno-associated virus] gene therapy[.]”
Following this news, Neurogene’s stock price fell over 44% on November 12, 2024.