Welcome to our dedicated page for Neurogene news (Ticker: NGNE), a resource for investors and traders seeking the latest updates and insights on Neurogene stock.
Neurogene Inc. develops genetic medicines for rare neurological diseases, with recurring updates centered on NGN-401, its investigational AAV9 gene therapy for Rett syndrome. Company news covers the EXACT™ transgene regulation technology, intracerebroventricular delivery for CNS-targeted gene therapy, clinical data from NGN-401 studies, and regulatory designations granted by U.S., European, and U.K. agencies.
Other recurring Neurogene developments include financial results, corporate presentations, scientific conference participation, board and executive appointments, and equity inducement grants under Nasdaq Listing Rule 5635(c)(4). The company also identifies NGN-101 as a conventional gene therapy development candidate for CLN5 Batten disease.
Neurogene (Nasdaq: NGNE) completed dosing in the Embolden™ registrational trial of NGN-401, a one-time gene therapy for Rett syndrome, dosing 25 participants versus an initial target of 20 and a pre-specified ITT population of up to 24.
NGN-401 at the 1E15 vg dose (n=35) has been generally well-tolerated with no hemophagocytic lymphohistiocytosis (HLH) reported. Primary analysis for a planned BLA will occur after 24 participants complete 12 months of follow-up, with topline data expected in 2H 2027. Updated Phase 1/2 interim data are planned for mid-2026.
Neurogene (Nasdaq: NGNE) approved inducement grants of non-qualified stock options covering 17,045 shares of common stock for two new employees under its 2025 Inducement Plan, effective June 1, 2026.
The options have an exercise price equal to the grant-date closing price and vest over four years.
Neurogene (Nasdaq: NGNE) reported first quarter 2026 results and clinical progress for NGN-401 in Rett syndrome.
About 90% of Embolden™ trial participants are dosed, NGN-401 at 1E15 vg has been generally well-tolerated with no HLH cases reported, and cash of $243.2 million is expected to fund operations into Q1 2028.
Neurogene (Nasdaq: NGNE) presented data at the ASGCT meeting supporting intracerebroventricular (ICV) delivery as a routine method for CNS-targeted gene therapies, including NGN-401 for Rett syndrome.
Preclinical and Phase 1/2 data showed broad brain biodistribution, functional gains in pediatric participants, and generally well-tolerated ICV administration.
Neurogene (Nasdaq: NGNE) announced inducement option grants under Nasdaq Listing Rule 5635(c)(4) dated May 5, 2026. The Compensation Committee approved non-qualified options to purchase an aggregate of 122,565 shares, including 120,000 shares issued to new Chief Commercial Officer Christy Shafer.
The awards were granted under the 2025 Inducement Plan, use the closing price on the grant date as the exercise price, and vest over four years with a vesting commencement date of April 20, 2026 for Ms. Shafer.
Neurogene (NASDAQ: NGNE) will present at ASGCT May 11-15, 2026 on intracerebroventricular (ICV) delivery as a deliberate neurosurgical route to maximize CNS gene therapy biodistribution.
Poster on May 12 (5:00–6:30 p.m. ET) will show preclinical data where ICV gave greater brain biodistribution than intrathecal‑lumbar, and clinical Phase 1/2 results reporting durable multidomain developmental gains and a safety profile at the 1E15 vg dose supporting advancement to the Embolden registrational trial.
Neurogene (Nasdaq: NGNE) announced two leadership moves on April 20, 2026: Christy Shafer was named Chief Commercial Officer to lead commercial strategy and launch readiness for NGN-401 for Rett syndrome, and Christine Mikail, J.D., was appointed to the Board to add strategic, financial, and operational oversight.
Shafer brings 20+ years launching rare-disease therapies; Mikail adds capital-formation and corporate-development experience and has supported Neurogene’s clinical and financial strategy.
Neurogene (Nasdaq: NGNE) announced inducement stock option grants totaling 69,140 shares to four new employees, approved April 6, 2026, under the company’s 2025 Inducement Plan.
The options have an exercise price equal to the closing price on the grant date and vest over four years (25% after one year, then 1/48 monthly).
Neurogene (Nasdaq: NGNE) reported full-year 2025 results and program updates on March 24, 2026. Key highlights: 100% enrollment in the Embolden registrational trial with >50% dosed and on track to complete dosing in Q2 2026. NGN-401 received FDA Breakthrough Therapy designation. Cash, cash equivalents and short-term investments were $269.0 million, expected to fund operations through Q1 2028. R&D expense was $75.0M and G&A was $28.3M for 2025; net loss was $90.4M. PPQ is planned mid-2026 to support BLA readiness.
Neurogene (Nasdaq: NGNE) management will participate in the Stifel 2026 Virtual CNS Forum in a fireside chat on Wednesday, March 18 at 9:00 a.m. ET. A live webcast and limited-time replay will be available in the Investor Relations events section on the company website.