Buy Rating Backed by Strong NGN-401 Clinical Progress, De-Risking Efficacy Data, and Solid Cash Runway in Rett Syndrome

Neurogene, the Healthcare sector company, was revisited by a Wall Street analyst yesterday. Analyst Paul Matteis from Stifel Nicolaus maintained a Buy rating on the stock and has a $46.00 price target.

Paul Matteis has given his Buy rating due to a combination of factors including clinical progress, safety signals, and regulatory momentum for NGN-401 in Rett syndrome. He highlights that enrollment in the pivotal EMBOLDEN trial is complete, more than half of patients have already been dosed without serious safety events such as HLH, and successful completion of dosing in 2Q would likely remove a key overhang on the stock.

He also points to encouraging early efficacy from the phase 1/2 study, where a substantial majority of treated patients have met both clinician-assessed improvement and functional milestone gains, with more mature 12‑month data expected in mid‑2026 to further de‑risk the program. In addition, he views recent regulatory interactions and receipt of Breakthrough Therapy designation, on top of prior RMAT and START designations, as reinforcing the strength of the program, and notes that Neurogene’s cash position of $269 million provides sufficient funding runway to execute on these milestones.

In another report released today, H.C. Wainwright also reiterated a Buy rating on the stock with a $70.00 price target.

Based on the recent corporate insider activity of 11 insiders, corporate insider sentiment is negative on the stock. This means that over the past quarter there has been an increase of insiders selling their shares of NGNE in relation to earlier this year.