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 (NGNE) is a clinical-stage biotechnology pioneer developing gene therapies for neurological disorders through its proprietary EXACT gene regulation platform. This page provides investors and researchers with timely updates on the company's scientific advancements, regulatory progress, and strategic partnerships.
Access consolidated news coverage including clinical trial milestones, research publications, and executive commentary. Stay informed about developments in gene therapy applications for rare neurological conditions, with updates vetted for accuracy and relevance.
Key content categories include treatment pipeline updates, FDA communications, collaborative research initiatives, and technology patent announcements. All materials maintain strict compliance with financial disclosure regulations while prioritizing accessibility for diverse audiences.
Bookmark this page for streamlined tracking of Neurogene's progress in advancing precision gene therapies. Check regularly for official press releases and third-party analysis of the company's groundbreaking work in neurological disease treatment.
Neurogene (Nasdaq: NGNE) reported Q2 2025 financial results and provided updates on its NGN-401 gene therapy program for Rett syndrome. The company has completed dosing in its Phase 1/2 trial and received FDA agreement on key elements of the Embolden™ registrational trial, with trial initiation activities already underway.
Financial highlights include a strong cash position of $274.5 million, providing runway into early 2028. Q2 2025 saw a net loss of $22.0 million, with R&D expenses of $19.4 million and G&A expenses of $6.7 million. The company plans to report updated clinical efficacy and safety data from the Phase 1/2 trial in H2 2025.
Neurogene (Nasdaq: NGNE), a clinical-stage company focused on genetic medicines for rare neurological diseases, has announced inducement grants for new employees. The Compensation Committee approved non-qualified stock options to purchase 27,480 shares of common stock for seven new employees on August 6, 2025.
The stock options, granted under the company's 2025 Inducement Plan, will vest over four years with 25% vesting on the first anniversary and the remaining vesting quarterly over 36 months. The exercise price equals the closing price of Neurogene's stock on the grant date. These grants comply with Nasdaq Listing Rule 5635(c)(4) as inducement material for employment.
Neurogene (Nasdaq: NGNE), a clinical-stage company focused on genetic medicines for rare neurological diseases, has announced inducement grants for two new employees. The Compensation Committee approved non-qualified stock options to purchase 4,290 shares of common stock on July 3, 2025.
The stock options, granted under the company's 2025 Inducement Plan, will vest over a four-year period, with 25% vesting after the first year and the remainder vesting quarterly over the following 36 months. The exercise price is set at the closing price of Neurogene's stock on the grant date. These grants comply with Nasdaq Listing Rule 5635(c)(4) and serve as employment inducements.
Neurogene (NASDAQ: NGNE) has announced key details of its Embolden™ registrational trial for NGN-401, a gene therapy treatment for Rett syndrome in females aged 3 years and older. The company has received FDA written agreement on critical aspects of the trial design.
The registrational trial will be a single-arm, open-label study with a proposed enrollment of 18 participants. The primary endpoint combines improvement in Clinical Global Impression-Improvement (CGI-I) scale and gain of developmental milestone/skill, compared to baseline. The study will use the 1E15 vector genomes (vg) dose.
In the Phase 1/2 trial, 10 participants have received treatment at the 1E15 vg dose, with no evidence of HLH/hyperinflammatory syndrome. Early data from the first four participants showed promising results, with participants collectively achieving 23 developmental milestones/skills across multiple domains.
The company reports a strong financial position with $292.6 million in cash and investments as of March 31, 2025, extending their runway into early 2028. Updated clinical efficacy and safety data are expected in the second half of 2025.
Neurogene (NASDAQ: NGNE) presented an evidence-based monitoring and treatment algorithm at the ASGCT Annual Meeting for detecting and treating hemophagocytic lymphohistiocytosis (HLH), a rare hyperinflammatory syndrome associated with high-dose AAV gene therapy. The protocol focuses on monitoring three key indicators (the "three Fs"): elevated ferritin levels, fever, and falling blood counts.
While HLH has been linked to AAV doses exceeding 1E14 vg/kg, Neurogene's Phase 1/2 trial of NGN-401 for Rett syndrome uses a lower dose in the E13 vg/kg range, where no HLH cases have been reported. The treatment algorithm includes high-dose corticosteroids as first-line treatment and anakinra as second-line treatment.
Neurogene (Nasdaq: NGNE) announced on May 8, 2025, that its Compensation Committee approved inducement grants for one new employee. The grants consist of non-qualified stock options to purchase 1,755 shares of common stock, issued under the company's 2025 Inducement Plan. The exercise price equals the closing price of Neurogene's stock on May 2, 2025. The options will vest over 4 years, with 25% vesting after the first year and the remainder vesting quarterly over the following 36 months, contingent on continued employment.
Neurogene has announced an upcoming oral presentation at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting in New Orleans, focusing on monitoring and treating hemophagocytic lymphohistiocytosis (HLH) in gene therapy patients.
Key highlights:
- The presentation will address HLH, a rare inflammatory condition linked to high-dose AAV gene therapy
- CEO Rachel McMinn emphasizes the importance of sharing effective approaches for monitoring and treating HLH
- The company's NGN-401 gene therapy trial for Rett syndrome uses a 1E15 vg dose level (E13 vg/kg range)
- No HLH cases have been reported at this dose level
Additional participation includes:
- A fireside chat on reviving hope in deprioritized cell and gene therapy programs
- A panel discussion on FDA's START Pilot Program insights
Neurogene (NASDAQ: NGNE) has published peer-reviewed data in Science Translational Medicine demonstrating the effectiveness of its EXACT™ transgene regulation technology in preclinical models of Rett syndrome. The study supports their ongoing Phase 1/2 clinical trial of NGN-401, their gene therapy candidate.
The research shows EXACT's capability to regulate gene therapy expression to therapeutic and tolerable levels, addressing a key challenge in treating Rett syndrome, which requires careful regulation of MeCP2 levels. The technology is designed to overcome limitations of conventional gene therapy, which can result in toxic levels of transgene expression.
In preclinical studies, NGN-401 demonstrated:
- Extended median survival in male knockout models
- Good tolerability across all doses in female murine models
- Well-tolerated results in non-human primates
- More controlled MECP2 mRNA levels compared to conventional gene therapy
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