Neurogene Inc. (NASDAQ: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, has commenced a public offering of shares of its common stock or, in lieu of common stock to certain investors that so choose, pre-funded warrants to purchase shares of its common stock. The company expects to grant the underwriters a 30-day option to purchase additional shares of its common stock at the public offering price, less underwriting discounts and commissions. The proposed offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed or as to the actual size or terms of the offering. All of the securities are being offered by Neurogene.

Neurogene intends to use the net proceeds from this offering, together with its existing cash, cash equivalents and marketable securities, to fund the ongoing clinical development of NGN-401. The funds will also support pre-commercial activities for NGN-401 and provide working capital for other general corporate purposes. NGN-401 is the company's lead gene therapy program, advancing as a potential best-in-class, one-time treatment for Rett syndrome.

Regulatory Filing and Underwriters

A shelf registration statement relating to these securities was filed with the Securities and Exchange Commission (SEC) on March 24, 2025, and was declared effective on April 4, 2025. This offering will be made only by means of a written prospectus, including a prospectus supplement, forming a part of an effective registration statement.

Leerink Partners, Stifel, Guggenheim Securities, LifeSci Capital and William Blair are acting as joint bookrunning managers for the proposed offering.

Offering Details

Neurogene Inc. announced updated, positive data from its Phase 1/2 clinical trial evaluating NGN-401 gene therapy for the treatment of females with Rett syndrome. The data demonstrated durable treatment effects, with 47 total developmental milestones gained across 10 participants and no plateau or milestone loss observed through 30 months of follow-up. The therapy was generally well-tolerated at the 1E15 vg dose as of the data cutoff date of June 16, 2026.

All participants showed clinical response, with 100% improving in CGI-I score and gaining at least one developmental milestone. The average gain was 4.7 milestones per participant, and all participants gained at least one milestone in the past 12 months. Developmental milestones were gained in a progressive, stepwise sequence, suggesting a restart of development post-treatment.

"We are encouraged by the long-term Phase 1/2 data across a broad age range and wide spectrum of disease severity, which we believe demonstrate that NGN-401 is driving gain of durable and clinically meaningful developmental milestones," said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene.

The EmboldenTM registrational trial has completed dosing with no treatment-related serious adverse events (SAEs) or dose-limiting toxicities (DLTs) as of June 16, 2026. Topline data from this trial are anticipated in the second half of 2027. Neurogene is set to host an investor and analyst webcast today at 8:00 a.m. ET to discuss the findings.

Key Clinical Data

Safety Profile

NGN-401 at the 1E15 vg dose remained generally well-tolerated (N=35) as of the data cutoff. No treatment-related SAEs or DLTs were reported in the registrational trial following the completion of dosing.