ARTICLE | Product Development
BioCentury’s Clinical Report also analyzes data from Syndax and Gan & Lee, plus pipeline updates from Sarepta, Tango and RAPT
By Lauren Martz, Executive Director of Biopharma Intelligence, and Paul Bonanos, Director of Biopharma Intelligence
November 13, 2024 12:47 AM UTC
Despite early signs of efficacy in a Phase I/II study of Neurogene’s gene therapy for Rett syndrome, the emergence of a treatment-related serious adverse event spooked investors, and the company appears to be moving only a lower dose forward in pediatric patients.
The safety signal resulted in an abrupt loss of value for Neurogene Inc. (NASDAQ:NGNE), whose shares slipped 44% to $40 on Tuesday, effectively completing a round trip this month. A $200 million PIPE announced Nov. 4 had helped propel the shares to a 52-week high of $74.49 on Friday, up 62% from their last close of $46.07 before the investment…
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- Source: https://www.biocentury.com/article/654170/adverse-event-casts-shadow-over-neurogene-s-rett-syndrome-readout