MDL-202

MDL-202 is an investigational AAV-mediated gene therapy developed by Modalis Therapeutics for the treatment of Myotonic Dystrophy Type 1 (DM1). It utilizes the proprietary CRISPR-GNDM (Guide Nucleotide-Directed Modulation) technology for epigenome editing. Unlike traditional CRISPR systems that create double-stranded breaks in DNA, CRISPR-GNDM employs a catalytically inactive Cas9 (dCas9) protein fused to a transcriptional repressor to modulate gene expression without altering the underlying DNA sequence. MDL-202 specifically targets and silences the *DMPK* (Dystrophia Myotonica Protein Kinase) gene. By inhibiting the transcription of mutant *DMPK* mRNA containing toxic CUG repeats, the therapy is designed to prevent the sequestration of Muscleblind-like (MBNL) proteins, thereby restoring normal RNA splicing and alleviating the clinical manifestations of DM1. The program is currently in the IND-enabling stage of development.