MyoArete is developing newly identified utrophin upregulation therapies that treat all Duchenne Muscular Dystrophy patients.
Utrophin when upregulated, functionally substitutes for the missing dystrophin and has been validated in preclinical studies, but not as yet translated to patients. MyoArete will leverage newly described platforms developed by the founder’s laboratory at Penn that target post-transcriptional repression mechanisms to harness utrophin upregulation-based treatments.
Utrophin upregulation treatments are predicted to improve function, slow disease progression, and increase lifespan in all DMD patients, regardless of mutation status. In addition, toxicity due to immune reactions or rejection is not expected with MyoArete’s approach of increasing the expression of utrophin, a protein that is already present in muscle. Notably, no toxicities have been noted with utrophin upregulation in animal studies. The platform and products are protected by patents and PCT filings.