Neurogene (NGNE) Corporate presentation summary

Mission and approach

• Focus on developing genetic medicines for severe neurological diseases, driven by patient and family needs.

• Utilizes a biology-first design and the EXACT platform for precise transgene expression and delivery.

NGN-401 program overview

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

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

• Embolden registrational trial is aligned with FDA for a single trial covering ages ≥3 years.

• Early commercial-readiness activities are underway for a multi-billion-dollar market.

Rett syndrome background and market opportunity

• Rett syndrome is a rare, progressive neurodevelopmental disorder caused by MECP2 gene variants.

• Affects 15,000–20,000 patients in major markets (US, EU, UK); no disease-modifying treatments exist.

• High burden on families and healthcare; payors are receptive to reimbursement for meaningful functional improvements.