Shape Therapeutics
Facts
- HQ
- Seattle
- Sectors
- Biotechnology, Gene therapy, RNA therapeutics, AAV delivery, Life sciences
- Founded
- 2018 Seattle business license
- Type
- Corporation Seattle business license
- Registered name
- Shape Therapeutics Inc Seattle business license
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.