Neurogene (NGNE) Corporate presentation summary

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.