Clinical EfficacyRegistrational expansion cohort results demonstrated robust dystrophin expression and consistent functional improvements, which materially strengthen the case for the lead DMD program's clinical success.
Regulatory PathwayManagement plans to submit for U.S. Accelerated Approval with an on-track regulatory filing in the second quarter, positioning the program for a potential expedited review and earlier market entry.
Strategic Value And M&A InterestStrong clinical and platform data have validated the company's delivery technology, increasing strategic interest and supporting the possibility of a premium acquisition given recent sector deal activity.