Shape Therapeutics and Rett Syndrome Research Trust Announce Partnership to Advance a One-Time AI-Designed RNA Editing Therapy for Rett Syndrome
Seattle-based Shape Therapeutics announced a partnership with the Rett Syndrome Research Trust (RSRT) to advance SHP-401, an investigational one-time gene therapy for Rett syndrome. Under the agreement, RSRT will fund studies evaluating Shape’s RNAfix technology, which is designed to correct the MECP2 R168X mutation associated with Rett syndrome.
Using a proprietary generative AI model, Shape designed an RNA payload that aims to correct the mutation at the RNA level and restore full-length MeCP2 protein. In a recent preclinical study, the company’s approach achieved approximately 70% RNA editing throughout the brain following a single intravenous dose in a mouse model. Through the new partnership, Shape and RSRT will evaluate the approach in non-human primates with the goal of generating data to support further development toward the clinic.
The partnership adds to the work underway in Washington to develop AI-enabled genetic medicines and move new approaches for rare diseases closer to clinical development.