FDA signs off on pivotal trial design for NGN-401 gene therapy
Neurogene received clearance from the FDA for a pivotal trial of NGN-401, which could support an application for the gene therapy's approval.| Rett Syndrome News
Neurogene received clearance from the FDA for a pivotal trial of NGN-401, which could support an application for the gene therapy's approval.| Rett Syndrome News
The first four Rett syndrome patients treated with the gene therapy candidate NGN-401 have seen meaningful gains in skills, data show.| Rett Syndrome News
NGN-401 is one of six picked, three each by the Center for Biologics Evaluation and Research, and the Center for Drug Evaluation and Research.| Rett Syndrome News
The US regulatory agency acts to support the potential gene therapy's development, with ongoing Phase 1/2 trial showing safety in patients.| Rett Syndrome News
