NGNE Stock: Insider Activity, Filings & Research
Neurogene Inc. (NGNE) — Drillr’s hub for NGNE insider activity, SEC filings, earnings signals and AI research. Over the trailing 3 months, NGNE insiders filed 0 open-market buys and 10 sales (SEC Form 4).
NGNE insider trading activity (SEC Form 4)
| Date | Insider | Type | Shares | Price |
|---|---|---|---|---|
| May 8, 2026 | Cvijic Christine Mikaildirector, officer: President and CFO | Sell | 4,800 | $30.07 |
| May 7, 2026 | Shafer Christinaofficer: Chief Commercial Officer | Grant | 120,000 | $28.94 |
| Mar 30, 2026 | McMinn Racheldirector, officer: Chief Executive Officer | Sell | 3,352 | $22.07 |
| Mar 30, 2026 | Cvijic Christine Mikailofficer: President and CFO | Sell | 2,558 | $21.62 |
| Mar 30, 2026 | Jordan Julieofficer: Chief Medical Officer | Sell | 828 | $21.89 |
| Mar 17, 2026 | Cobb Stuartofficer: Chief Scientific Officer | Sell | 148 | $22.08 |
| Mar 17, 2026 | Cvijic Christine Mikailofficer: President and CFO | Sell | 1,395 | $21.25 |
| Mar 17, 2026 | Cvijic Christine Mikailofficer: President and CFO | Sell | 263 | $22.02 |
| Mar 17, 2026 | Cvijic Christine Mikailofficer: President and CFO | Sell | 2,387 | $20.42 |
| Mar 17, 2026 | Cobb Stuartofficer: Chief Scientific Officer | Sell | 3,991 | $20.41 |
| Mar 17, 2026 | Cobb Stuartofficer: Chief Scientific Officer | Sell | 2,658 | $21.34 |
| Mar 3, 2026 | Cvijic Christine Mikailofficer: President and CFO | Sell | 4,800 | $25.11 |
| Feb 24, 2026 | BAFFI ROBERTdirector | Grant | 12,050 | $17.64 |
| Feb 24, 2026 | McMinn Racheldirector, officer: Chief Executive Officer | Grant | 155,100 | $17.64 |
| Feb 24, 2026 | McMinn Racheldirector, officer: Chief Executive Officer | Grant | 25,900 | — |
Source: NGNE SEC Form 4 filings, latest May 8, 2026. For informational purposes only — not investment advice.
Neurogene Inc. company profile
Overview
Neurogene Inc. (NASDAQ:NGNE) is a clinical-stage biotechnology company founded in 2014 and headquartered in New York, New York. The company specializes in developing gene therapies for rare neurological diseases, focusing on conditions that primarily affect children and have limited or no treatment options. Since its initial public offering in 2014, Neurogene has been advancing its pipeline of investigational treatments through clinical trials, with its lead programs targeting Rett syndrome and Batten disease.
Business
Neurogene operates in the gene therapy sector of biotechnology, which represents one of the most cutting-edge areas of modern medicine. Gene therapy involves introducing genetic material into a patient's cells to treat or prevent disease, essentially correcting defective genes or providing new cellular functions. The company uses adeno-associated virus (AAV) vectors as delivery vehicles to transport therapeutic genes into specific cells in the body. The company's pipeline focuses on two primary programs. NGN-401 is an investigational AAV9 gene therapy designed to treat Rett syndrome, a rare genetic neurological disorder that primarily affects girls and causes severe cognitive and physical disabilities. Rett syndrome is caused by mutations in the MECP2 gene, which leads to problems with brain development and function. NGN-401 aims to deliver a functional copy of the MECP2 gene to neurons in the brain. NGN-101 targets neuronal ceroid lipofuscinosis subtype 5 (CLN5 Batten disease), another rare genetic disorder that causes progressive neurodegeneration in children. Batten disease is characterized by the accumulation of toxic substances in cells due to enzyme deficiencies, leading to vision loss, seizures, and cognitive decline. This therapy seeks to provide the missing or defective CLN5 gene to restore normal cellular function. Both conditions represent significant unmet medical needs with no approved gene therapies currently available, making Neurogene's programs potentially first-in-class treatments for these devastating childhood diseases.
Revenue model
As a clinical-stage biotechnology company, Neurogene does not yet generate meaningful revenue from product sales. The company's minimal revenue of $925,000 in 2024 likely comes from research collaborations, grants, or licensing arrangements rather than commercial product sales. The company's future business model will depend on successfully advancing its gene therapies through clinical trials and ultimately gaining regulatory approval. Once approved, gene therapies typically command premium pricing due to their one-time treatment potential and the severe nature of the diseases they address. Gene therapies for rare diseases often receive orphan drug designations, which provide market exclusivity and other regulatory advantages. The target customers would be specialized medical centers, children's hospitals, and healthcare systems that treat patients with these rare neurological conditions. Several factors could significantly impact Neurogene's future margins and commercial success. Positive clinical trial results demonstrating safety and efficacy would be the primary driver of value creation. Regulatory approval pathways for gene therapies, while specialized, can be expedited for rare diseases through programs like breakthrough therapy designation. Manufacturing costs for AAV-based gene therapies are typically high due to complex production requirements, but the one-time treatment nature allows for premium pricing. Competition from other gene therapy developers targeting the same diseases could affect market share and pricing power. Additionally, reimbursement decisions by insurance providers and healthcare systems will be crucial for commercial success, as gene therapies often require substantial upfront investments despite their potential long-term cost savings.
Competitive moat
Neurogene's competitive position is primarily built on its specialized expertise in neurological gene therapies and its clinical development programs for specific rare diseases. The company's potential moat lies in several factors: first-mover advantage in targeting specific genetic subtypes of rare neurological diseases, specialized manufacturing and delivery capabilities for AAV-based therapies, and the significant regulatory and clinical development barriers that competitors would need to overcome. However, the company's moat is relatively narrow and still developing. The gene therapy space is highly competitive, with numerous well-funded biotechnology companies and pharmaceutical giants pursuing similar approaches. Larger competitors like Roche, Novartis, and other established players have greater resources for clinical development, manufacturing scale, and regulatory expertise. The scientific and technical barriers, while substantial, are not insurmountable for well-resourced competitors. The company's intellectual property position and clinical data will be crucial in establishing a defensible competitive position. Success in clinical trials could provide significant first-mover advantages, especially given the small patient populations for these rare diseases. However, if clinical trials fail or show limited efficacy, the company would face substantial challenges in maintaining its competitive position. The ultimate strength of Neurogene's moat will depend on successfully executing its clinical programs and establishing itself as a leader in treating these specific rare neurological conditions.
Risks & safety
Neurogene presents a mixed margin of safety profile typical of clinical-stage biotechnology companies, with strong liquidity but inherent execution risks. • Liquidity position: Strong cash position of $70.8 million as of Q1 2025, down from $136.6 million at year-end 2024, indicating significant cash burn • Cash burn rate: Quarterly operating cash outflow of approximately $20.8 million in Q1 2025, suggesting roughly 3-4 quarters of runway at current burn rate • Debt levels: Minimal debt with debt-to-equity ratio of 0.046, indicating low financial leverage • Current ratio: Exceptionally strong at 21.0, demonstrating excellent short-term liquidity • Valuation metrics: Trading at 0.84x book value, suggesting potential undervaluation relative to net assets • Revenue generation: Essentially pre-revenue with minimal income, creating dependency on clinical trial success and future funding • Solvency risk: Low immediate solvency risk due to strong balance sheet, but will require additional funding within 12-18 months to continue operations
Recent development
Based on the available financial data, Neurogene has been primarily focused on advancing its clinical programs for NGN-401 and NGN-101 through the development pipeline. The company's research and development expenses have remained consistently high, reflecting ongoing clinical trial activities and regulatory preparation work. The company completed what appears to be a significant financing round between Q3 2024 and Q4 2024, as evidenced by the substantial increase in cash and short-term investments from $66.6 million to $136.6 million. This capital raise likely supports continued clinical development activities and extends the company's operational runway. The minimal revenue of $925,000 reported in 2024 suggests the company may have entered into research collaborations or received milestone payments, though the primary focus remains on advancing its proprietary gene therapy programs. The consistent quarterly cash burn of approximately $15-21 million indicates steady progress in clinical development activities, manufacturing preparations, and regulatory interactions necessary for advancing gene therapies through the approval process.
NGNE company profile · for informational purposes only — not investment advice.
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