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. (Nasdaq: NGNE) generates news primarily around its gene therapy programs for rare neurological diseases, with a central focus on NGN-401 for Rett syndrome. As a clinical-stage company, Neurogene regularly issues updates on clinical trial progress, interim data, regulatory interactions and corporate developments.
Recent press releases highlight multiple milestones for NGN-401, including dosing of participants in the Embolden registrational trial, initiation of numerous U.S. clinical trial sites, and positive interim data from the Phase 1/2 trial in pediatric and adolescent/adult cohorts. These updates describe multidomain, durable functional gains and developmental milestone acquisition in females with Rett syndrome, along with safety observations at the 1E15 vg dose.
Visitors to this NGNE news page can review announcements on trial design for Embolden, preclinical data comparing intracerebroventricular and intrathecal lumbar delivery, and regulatory designations from the FDA, EMA and UK MHRA. Neurogene also reports on participation in healthcare and genetic medicines conferences, where management presents company overviews and clinical data, as well as on financial results and cash runway disclosures through quarterly earnings releases.
Additional news items cover corporate matters such as inducement grants under Nasdaq Listing Rule 5635(c)(4) and updates furnished via Form 8-K. For investors and followers of Rett syndrome research, the NGNE news stream provides a consolidated view of Neurogene’s clinical progress, regulatory status and operational execution around NGN-401 and its broader gene therapy platform over time.
Neurogene (Nasdaq: NGNE) announced the FDA granted Breakthrough Therapy designation for NGN-401 for Rett syndrome, based on interim Phase 1/2 data through Oct 30, 2025 showing clinically meaningful, durable, multidomain functional improvements.
NGN-401 is a one-time, intracerebroventricular gene therapy using EXACT transgene regulation; dosing in the Embolden registrational trial is expected to complete in Q2 2026. The program also holds RMAT and Rare Pediatric Disease designations and participated in FDA’s START Pilot Program.
Neurogene (Nasdaq: NGNE) management will participate in three investor conferences in February–March 2026: Guggenheim Emerging Outlook: Biotech Summit (Feb 12, 9:00 a.m. ET), TD Cowen 46th Annual Health Care Conference (Mar 4, 10:30 a.m. ET), and Leerink Global Healthcare Conference (Mar 9, 1:40 p.m. ET).
Each appearance includes a fireside chat and investor meetings. Live webcasts and limited-time replays will be available via the Investor Relations events page on Neurogene's website.
Neurogene (Nasdaq: NGNE) announced an inducement grant of non-qualified stock options totaling 2,820 shares to one new employee, approved by the Compensation Committee on February 2, 2026. The grant was made under the company’s 2025 Inducement Plan per Nasdaq Listing Rule 5635(c)(4).
The options have an exercise price equal to the closing stock price on the grant date and vest over four years: 25% at the first anniversary of vesting commencement, then monthly vesting of 1/48 thereafter, subject to continued employment.
Neurogene (NASDAQ: NGNE) provided a program update for NGN-401, a gene therapy for Rett syndrome, reporting that multiple participants were dosed in the Embolden registrational trial in Q4 2025 and that dosing completion is expected in Q2 2026. The company initiated 13 U.S. Embolden sites and reported positive interim Phase 1/2 data showing durable multidomain improvements across disease severity. Neurogene plans to present interim pediatric and adolescent/adult cohort results (pediatric n=8; adolescent/adult n=2) by mid-2026 with ≥12 months follow-up, is advancing early commercial-readiness and payor engagement, and expects cash runway through Q1 2028 while targeting a BLA submission as a key milestone.
Neurogene (Nasdaq: NGNE) announced management will participate in the 44th Annual J.P. Morgan Healthcare Conference on Wednesday, January 14 with a company overview presentation and investor meetings starting at 7:30 a.m. PT.
A webcast of the presentation will be available from the company's Investor Relations website under events, and a replay will be accessible for a limited time.
Neurogene (Nasdaq: NGNE) announced that its Compensation Committee approved inducement grants of non-qualified stock options totaling 4,860 shares to two new employees, with a grant date of December 2, 2025.
The options were issued under the company’s 2025 Inducement Plan in accordance with Nasdaq Listing Rule 5635(c)(4). The exercise price equals the closing price of NGNE common stock on the grant date. Vesting occurs over four years: 25% on the first anniversary of the vesting commencement date and 1/48 of the award each month thereafter, subject to continued employment.
Neurogene (Nasdaq: NGNE) reported third quarter 2025 results and program updates on November 14, 2025. The company reported $265.4 million in cash, cash equivalents and short-term investments as of September 30, 2025, which it says funds operations through the first quarter of 2028. Clinical updates highlighted updated interim Phase 1/2 pediatric data for NGN-401 in Rett syndrome showing multidomain, durable gains with an aggregate 35 developmental milestones/skills gained across 8 pediatric participants as of an October 30, 2025 cutoff.
Neurogene dosed the first participant in the Embolden registrational trial and has initiated 12 of 13 sites with complete enrollment expected in 3–6 months. Preclinical data presented support intracerebroventricular (ICV) delivery for broader brain biodistribution versus intrathecal lumbar (IT-L).
Neurogene (Nasdaq: NGNE) reported positive interim Phase 1/2 data for its NGN-401 gene therapy in the pediatric Rett syndrome cohort (data cutoff Oct 30, 2025). All 8 pediatric participants showed functional gains across core domains with an aggregate 35 developmental milestones/skills gained and durable, multidomain improvements observed up to 24 months post treatment. Safety (N=10) at the 1E15 vg dose was generally well-tolerated; treatment-related adverse events were Grade 1–2 and no HLH was observed. Additional clinical data is planned for 2026, and management will discuss results at the Stifel 2025 Healthcare Conference webcast on Nov 12, 2025 at 4:40 p.m. ET.
Neurogene (Nasdaq: NGNE) announced that the first participant was dosed in the Embolden registrational trial of NGN-401 for Rett syndrome on November 6, 2025. Embolden is a single-arm, open-label, baseline-controlled study evaluating a 1E15 vg intracerebroventricular dose in 20 females ages ≥3.
The company initiated 12 of 13 U.S. sites and expects to complete enrollment within three to six months. Management will present Phase 1/2 interim data at the Stifel 2025 Healthcare Conference on November 12, 2025, with a webcast and temporary replay available via investor relations.
Neurogene (Nasdaq: NGNE) announced an inducement equity grant approved by its Compensation Committee on November 4, 2025. The company granted non-qualified stock options to purchase 1,185 shares to one newly hired employee under the 2025 Inducement Plan in accordance with Nasdaq Listing Rule 5635(c)(4).
The options have an exercise price equal to the closing price on the grant date and vest over four years: 25% at the first anniversary of the vesting commencement date and the remaining 75% in equal installments every three months over the following 36 months, subject to continued employment.