Neurogene Inc. (NGNE)

Neurogene's stock experienced fluctuations, including a price drop in November 2024 due to a serious adverse event in a NGN-401 trial, followed by FDA clearance to continue testing at a lower dose. Positive regulatory updates for NGN-401 were announced in October 2025, with plans to initiate the Embolden™ Registrational Trial in Q4 2025. The company reported increased net losses in Q2 2025 due to higher research and development expenses. The stock's 52-week low was $6.875 on April 8, 2025, and it has since risen significantly.

The demand for Neurogene, Inc.'s products and services is not typically subject to seasonal fluctuations. Demand is primarily driven by the prevalence of the rare diseases it targets and the progress and success of its clinical trials.

Neurogene Inc. is a clinical-stage biotechnology company focused on developing genetic medicines for rare neurological diseases, operating within the Healthcare sector, specifically in the Biotechnology industry. Key product candidates include NGN-401, in Phase 1/2 clinical trials for Rett syndrome, and NGN-101, in Phase 1/2 clinical trials for CLN5 Batten disease. The company uses its EXACT transgene regulation technology to deliver therapeutic levels of treatment while mitigating transgene toxicity.

Neurogene Inc. is headquartered in New York, New York, and maintains a gene therapy manufacturing facility in Houston, Texas, supporting GMP production of NGN-401 and future clinical development activities.

Neurogene's brand reputation has been influenced by its clinical trial progress and related events. The adverse event in the NGN-401 trial in November 2024 posed a challenge, leading to a stock price drop. However, the subsequent FDA clearance and plans to advance NGN-401 into a registrational trial in Q4 2025 demonstrate a commitment to patient safety. Neurogene's participation in scientific and investor conferences also contributes to its visibility.

Neurogene Inc. has a mix of institutional, insider, and retail ownership. Redmile Group LLC is the largest individual shareholder, holding 27.09% of the company's shares, valued at approximately $112.25 million. Other notable individual owners include Jonathan G. Drachman and Gary Bridger.