Sep 11, 2026 —
Exegenesis Bio and Modalis Therapeutics have signed a research and license agreement to develop MDL-201, a candidate for Duchenne muscular dystrophy (DMD).
MDL-201 pairs Modalis’ CRISPR-GNDM (Guide Nucleotide-Directed Modulation) technology, which uses a modified, non-cutting CRISPR system to tune gene expression, with Exegenesis Bio’s engineered muscle-tropic adeno-associated virus (AAV) capsid, EMC181. The goal is to raise levels of utrophin, a naturally occurring protein that can stand in for the dystrophin missing in DMD, an approach that would apply regardless of a patient’s specific mutation.
DMD is a severe, progressive muscle-wasting disease caused by mutations in the dystrophin gene.
