Neurogene (NGNE) investor relations material

Company overview and lead program

• Focuses on genetic medicines for severe neurological diseases, with NGN-401 targeting Rett syndrome, a debilitating disorder with significant unmet need affecting 15,000–20,000 patients in the US and Europe.

• NGN-401 is an AAV gene therapy designed as a one-time treatment to address the underlying cause of Rett syndrome, showing substantial functional improvement in phase I/II data with 100% patient response.

• Completed dosing for the registrational Embolden trial in Q2, with primary endpoint data expected in the second half of 2027.

Technology and trial design

• NGN-401 uses the full-length MECP2 gene and incorporates EXACT, a microRNA-based feedback loop to control transgene expression and avoid toxicity.

• Intracerebroventricular (ICV) delivery was chosen to maximize brain neuron exposure, differentiating it from competitors.

• The Embolden trial uses a baseline-controlled design, measuring developmental milestone gains and clinician-assessed improvement over 12 months.

Efficacy and patient outcomes

• Phase I/II data show patients regaining or acquiring developmental milestones rarely seen after regression in Rett syndrome.

• Benefits observed across a diverse patient group, with no evidence that only certain subgroups respond.

• The trial design leverages natural history data to benchmark patient progress and address disease heterogeneity.