Neurogene hits a setback in the clinic, days after fundraise

BioPharma Dive November 12, 2024
Ned Pagliarulo

The gene therapy developer disclosed that a participant in one of its clinical trials experienced a serious side effect, marring results that analysts otherwise saw as positive.

Dive Brief:

• Shares in gene therapy developer Neurogene fell by more than 40% Tuesday after the company disclosed it had learned a participant given one of its experimental treatments in a clinical trial experienced a serious side effect.
• The disclosure was made alongside results from the first few patients treated with a low dose of the treatment, called NGN-401 and designed to address the neurodevelopmental disorder Rett syndrome. Neurogene said it was made aware on Tuesday of the side effect, which occurred in the third patient given a higher dose.
• Separately, Neurogene...

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