Neurogene Reports Positive Long-Term Data for NGN-401 Gene Therapy in Rett Syndrome
In Neurogene's Phase 1/2 trial, every participant improved on the CGI-I scale and gained at least one developmental milestone, averaging 4.7 milestones per participant, with no plateau or milestone loss observed as of the data cutoff. NGN-401 has remained generally well tolerated.
Neurogene also completed dosing in its Embolden™ registrational trial ahead of its enrollment target and has begun manufacturing validation work to support a potential BLA submission.
