ORB-101

ORB-101 is a preclinical circular RNA (oRNA) therapy being developed for the treatment of Duchenne Muscular Dystrophy (DMD). Developed by Orbital Therapeutics, which was acquired by Bristol Myers Squibb in late 2025, the candidate utilizes a proprietary circular RNA platform designed to provide durable and stable expression of a functional dystrophin protein in muscle cells. Unlike traditional linear mRNA, circular RNA is engineered to be more resistant to exonuclease degradation, potentially allowing for higher and more sustained therapeutic protein levels with less frequent dosing. The therapy aims to address the underlying cause of DMD, a progressive muscle-wasting disease caused by the lack of functional dystrophin, by restoring protein function in muscle tissue.