Shape Therapeutics and Rett Syndrome Research Trust partner to advance SHP-401 AI-designed RNAfix® therapy correcting MECP2 R168X toward IND studies

Shape Therapeutics and the Rett Syndrome Research Trust partnered to fund translational studies of SHP-401, an intravenously delivered, one-time RNAfix® gene therapy using AI-designed guide RNA to correct the MECP2 R168X mutation for Rett syndrome. Preclinical mouse data posted to bioRxiv show ~70% RNA editing in the brain and restoration of full-length MeCP2 after a single IV dose, supporting IND-enabling study goals in non-human primates.