Neurogene’s NGN-401 gene therapy scores well in Rett syndrome

New York, USA-based rare neurological disease drug developer Neurogene (Nasdaq: NGNE) has announced positive interim clinical data in the first four participants in the low-dose cohort of its ongoing Phase I/II open-label trial designed to evaluate NGN-401 gene therapy for the treatment of female pediatric patients with Rett syndrome. Low-dose NGN-401 has demonstrated a favorable safety profile.

Investors, however, were not impressed, sending the firm’s share plunging 44% to $40.00 by close of trading, as Neurogene also disclosed that it became aware yesterday that the third high-dose participant, who was recently dosed, experienced a treatment-related serious adverse event (AE) described as "consistent with known risks of AAV gene therapy."