Neurogene (NGNE) Corporate presentation summary

Mission and approach

• Focus on developing life-changing genetic medicines for severe neurological diseases.

• Utilizes a biology-first design and precision delivery for maximum drug distribution.

• EXACT platform enables precise transgene expression tailored to patient needs.

NGN-401 program highlights

• NGN-401 is advancing as a potential one-time, transformative gene therapy for Rett syndrome.

• Demonstrated durable, multidomain improvements across all disease severities.

• Received multiple FDA designations, including Breakthrough Therapy and RMAT.

• Embolden registrational trial completed dosing (N=25), exceeding targets due to high demand.

• Topline data from Embolden expected in 2H 2027; BLA-enabling PPQ runs to finish by end of 2026.

Unmet need and market opportunity

• Rett syndrome has no approved disease-modifying therapies; current care is lifelong and symptom-focused.

• Estimated 15,000–20,000 patients in US, EU, and UK, with ~6,000 diagnosed in the US.

• Patient population spans all ages, creating broad adoption potential for transformative therapies.

• NGN-401 targets the root genetic cause, aiming to restart developmental progression.