Clinical TrialsInitial data shows all four patients achieved a clinically meaningful rating of 'much improved' and a collective achievement of 23 developmental milestones/skills.
Financial StabilityCash reserves at the end of the period were $274.5M, expected to provide financial runway into early 2028, indicating strong financial planning.
Regulatory ProgressNeurogene has received clearance to enroll patients as young as three years of age, which would be a potential commercial advantage.