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Neurogene
Neurogene (NGNE) investor relations material
Neurogene Guggenheim Securities Emerging Outlook: Biotech Summit 2026 summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Guggenheim Securities Emerging Outlook: Biotech Summit 2026 summary12 Feb, 2026
Company overview and lead program
Focuses on genetic medicines for rare neurologic diseases using a biology-first approach.
Lead program NGN-401 targets Rett syndrome, a multi-billion dollar market with 15,000–20,000 patients in the US and Europe.
NGN-401 pivotal study began dosing in Q4 2025, with enrollment and dosing completion expected in Q2 2026.
Phase I/II data showed 35 milestones gained across 8 participants, indicating multi-domain, durable improvements.
Upcoming catalyst is the completion of enrollment and 12-month data readout for all 10 patients in mid-2026.
Differentiation and technology
NGN-401 uses a full-length human MECP2 gene and proprietary technology to regulate expression, minimizing toxicity.
Delivery method ensures drug reaches neurons in the brain, addressing the root cause of Rett syndrome.
Route of administration (ICV) is supported by caregivers, KOLs, and payers, who prioritize efficacy over delivery concerns.
Payers focus on clear, objective efficacy endpoints, such as milestone gains.
Safety, dosing, and monitoring
Previous HLH event at higher dose (3E15); current pivotal dose (1E15) is three times lower and has shown no HLH cases.
Monitoring protocol includes ferritin and other immune markers for early detection and reversibility of adverse events.
Nerve conduction issues observed were transient and align with known AAV gene therapy class effects.
Safety updates will be disclosed if material events occur; current data shows no new safety concerns.
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Next Neurogene earnings date
TD Cowen 46th Annual Health Care Conference4 Mar, 2026
Neurogene
Next Neurogene earnings date
TD Cowen 46th Annual Health Care Conference4 Mar, 2026
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Frequently asked questions
Neurogene Inc. (NGNE) is a clinical-stage biotechnology company focused on developing genetic medicines for rare neurological diseases. Utilizing its proprietary transgene regulation technology, known as Expression Attenuation via Construct Tuning (EXACT), Neurogene aims to address high unmet medical needs that are not effectively treated by conventional gene therapies. The company is headquartered in New York, New York, and its shares are listed on the NASDAQ.
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