Research 🇬🇧 Important for Genetic Diseases generally

Rett Syndrome Gene Therapy Trial Shows Early Developmental Gains

person Neurogene

calendar_today Jun 29, 2026

Rett syndrome gene therapy candidate NGN-401 showed early Phase 1/2 trial results in June 2026. The one-time treatment delivers functional MECP2 through intracerebroventricular administration. In 10 participants, most showed developmental gains and improved CGI-I scores. Some milestones required a post-treatment restart, but reported gains remained durable, with no participant losing skills gained during the trial.