SHP-401

SHP-401 is an investigational, one-time RNA-editing gene therapy developed by Shape Therapeutics for the treatment of Rett syndrome. It is designed to target and correct the MECP2 R168X mutation, which is the most common causal point mutation in Rett syndrome, accounting for approximately 10% of cases. SHP-401 utilizes Shape's proprietary RNAfix guide RNA platform, where an AI-designed guide RNA recruits native adenosine deaminase acting on RNA (ADAR) enzymes to correct the R168X mutation at the messenger RNA (mRNA) level, thereby restoring the production of full-length, functional methyl-CpG-binding protein 2 (MeCP2) without permanently altering the DNA. The therapy is delivered intravenously using a proprietary blood-brain barrier (BBB)-penetrant, adeno-associated virus 5 (AAV5)-derived capsid.