MDL-103

MDL-103 is an investigational AAV-based gene therapy developed by Modalis Therapeutics for the treatment of facioscapulohumeral muscular dystrophy (FSHD). It utilizes Modalis's proprietary CRISPR-GNDM technology to epigenetically silence the DUX4 gene, which is aberrantly activated in FSHD and drives disease pathology. Unlike traditional genome-editing approaches that introduce double-strand DNA breaks, MDL-103 modulates gene expression without cutting DNA, aiming for durable suppression of DUX4 with muscle-specific delivery via an adeno-associated virus (AAV) vector. Preclinical studies have demonstrated promising efficacy in mouse models and safety in non-human primates[1][2][4][7][10].

Other names

MDL 103MDL103MDL-103CRISPR-GNDM gene editing therapy