Neurogene reports positive interim efficacy data from low-dose cohort of NGN-401 gene therapy for Rett syndrome

Neurogene Inc.

All 4 low-dose participants achieved a 2-point improvement on clinician-rated CGI-I scale from baseline.
Caregiver-completed RSBQ scores improved 28–52% from baseline; participants acquired developmental milestones not seen in natural history.
Low-dose NGN-401 well-tolerated with no treatment-related SAEs; emerging SAE noted in a third high-dose participant.
Initiated adolescent/adult cohort (age ≥16) and gained FDA alignment on potency assay and manufacturing scale-up for registrational trial.
Company discontinued CLN5 Batten disease program after RMAT application denial; evaluating options.

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