Neurogene expands Rett syndrome gene therapy trial, adds high-dose cohort

Neurogene Inc.

Cohort 1 expanded from 5 to 8 patients; staggered dosing removed for parallel enrollment.
New Cohort 2 with higher dose (3×10^15 vector genomes) added; first patient dosing expected Q2 2024.
Third patient dosed in Cohort 1; no serious adverse events or overexpression-related toxicity observed.
Interim Cohort 1 data expected Q4 2024; Cohort 2 data expected H2 2025.
Targeted immunosuppression (rituximab, sirolimus) added for Cohort 2 to prevent AAV immune responses.

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