MDL-207

MDL-207 is a preclinical-stage gene therapy being developed by Modalis Therapeutics for the treatment of Dravet syndrome, a severe and rare form of childhood epilepsy. The therapy utilizes Modalis's proprietary CRISPR-GNDM (Guide Nucleotide-Directed Modulation) epigenome editing technology to upregulate the expression of the SCN1A protein. Dravet syndrome is primarily caused by haploinsufficiency of the SCN1A gene, which encodes the alpha subunit of the voltage-gated sodium channel Nav1.1. Unlike traditional CRISPR gene editing that involves cutting DNA, CRISPR-GNDM employs a catalytically inactive Cas9 (dCas9) to modulate gene expression through epigenetic modification without introducing double-strand DNA breaks. This approach aims to restore physiological levels of the deficient protein to address the underlying molecular cause of the disease.