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 precision delivery for maximum drug distribution.

• Employs the EXACT platform for precise transgene expression.

NGN-401 for Rett syndrome: clinical progress and design

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

• Received FDA Breakthrough Therapy, RMAT, and other regulatory designations.

• Embolden registrational trial aligned with FDA, targeting broad age range (>3 years); dosing nearly complete.

• Phase 1/2 trial showed durable, multidomain improvements across disease severity.

• 100% of participants showed functional improvements; 88% improved CGI-I scores; no plateau up to 24 months.

Disease 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); 1:10,000 females worldwide.

• No disease-modifying treatments exist; current care is lifelong and symptom-focused.

• High burden on families and healthcare; payors value meaningful functional improvements.