On Thursday, Neurogene Inc. (NASDAQ:NGNE) received an Outperform rating from William Blair, with a price target set at $61.00. The coverage initiation is based on the potential of Neurogene's lead gene therapy candidate, NGN-401, for the treatment of Rett syndrome, a rare genetic neurological disorder.
William Blair's analysis suggests that NGN-401, which includes a full-length human MECP2 transgene and Neurogene's proprietary EXACT technology, may offer regulated and consistent gene expression. This approach aims to minimize the risks of gene overexpression and the toxicities associated with it.
NGN-401 is currently undergoing a Phase I/II trial, which is particularly notable for its direct administration method that could enhance biodistribution in the brain, potentially leading to improved clinical outcomes.
The firm highlights the U.S. Food and Drug Administration's (FDA) decision to allow Neurogene to expedite its clinical trial by directly enrolling pediatric patients, bypassing the usual adult dosing studies. This move is seen as a testament to the strength of NGN-401's preclinical safety data.
Treating pediatric patients early is considered advantageous as they are more likely to gain maximum therapeutic benefits, which could lead to a robust dataset for the company.
The anticipation around the initial data from the Phase I/II trial of NGN-401, expected in the fourth quarter of 2024, is identified as a potential key catalyst for Neurogene's stock.
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