EVX-Rett

EVX-Rett is a preclinical exosome-based gene replacement therapy being developed by Evox Therapeutics for the treatment of Rett Syndrome. The program utilizes Evox's proprietary DeliverEX platform to deliver CRISPR/Cas9 components encapsulated within exosomes to target and repair or replace the MECP2 gene. Rett Syndrome is a rare, severe neurodevelopmental disorder primarily caused by mutations in the Methyl-CpG binding protein 2 (MECP2) gene, leading to a deficiency in functional MECP2 protein. By leveraging exosome technology, EVX-Rett aims to achieve efficient delivery across the blood-brain barrier to restore MECP2 expression in the central nervous system, potentially addressing the underlying genetic cause of the disease.

Other names

Rett Syndrome program