X-0112

X-0112 is a synthetic DNA-binding pyrrole-imidazole (PI) polyamide being developed by Triplex Therapeutics for the treatment of Duchenne Muscular Dystrophy (DMD). As a PI polyamide, it is a small molecule designed to recognize and bind to specific sequences in the minor groove of double-stranded DNA. Unlike exon-skipping therapies that produce a truncated, partially functional dystrophin protein, X-0112 aims to preserve the full length of the dystrophin gene and enable the production of more functional, full-length dystrophin protein. This approach is intended to provide a more effective therapeutic or potentially curative option for patients with DMD. The drug is currently in the discovery and preclinical stages of development.