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Neurogene
Neurogene (NGNE) investor relations material
Neurogene Corporate presentation summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Corporate presentation summary12 Jan, 2026
Mission and approach
Focus on developing genetic medicines for severe neurological diseases using a biology-first design and precision delivery for maximum drug distribution.
EXACT platform enables precise transgene expression, driven by patient and family needs.
NGN-401 program highlights
NGN-401 is advancing as a potential one-time, transformative gene therapy for Rett syndrome, showing durable multidomain improvements across disease severity.
Embolden registrational trial, aligned with FDA, targets broad age range (≥3 years) with dosing underway and completion expected in 2Q'26.
Early commercial-readiness activities are in progress for a multi-billion-dollar market.
Clinical and regulatory progress
Completed Phase 1/2 trial enrollment and dosed multiple participants in Embolden trial.
Positive interim Phase 1/2 data reported; received PRIME designation from EMA.
FDA's potential impact on Embolden trial design?
Outpatient pathway's impact on NGN-401 reimbursement?
How does EXACT prevent MeCP2 overexpression?
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Next Neurogene earnings date
44th Annual J.P. Morgan Healthcare Conference14 Jan, 2026
Neurogene
Next Neurogene earnings date
44th Annual J.P. Morgan Healthcare Conference14 Jan, 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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