One patient under 4 years old dosed with RGX-202 achieved microdystrophin expression at 122.3% of control. How clinically meaningful is this level of expression in very young patients, and what could it suggest about the therapy's disease-modifying potential at an early stage of muscle degeneration?

RGX-202 has reported the highest vector genome copies across investigational and approved gene therapies. How important is vector copy number in the context of transgene expression, and what might this suggest about RGX-202’s efficiency in muscle tissue targeting?

The data confirms RGX-202 microdystrophin appropriately localizes to the sarcolemma. From your perspective, why is correct localization a critical factor in gene therapy efficacy, and how does this support functional muscle restoration?

REGENXBIO is the only sponsor currently dosing patients under 4 years old in the U.S. What are the unique risks and benefits of intervening this early, and could this redefine the standard of care for Duchenne in this underserved population?

The absence of serious adverse events or AESIs is encouraging. How does the proactive short-course immune modulation regimen used in this trial contribute to the overall tolerability, and do you think this sets a new bar for gene therapy protocols in DMD?

With biomarker data now indicating high and consistent microdystrophin levels, how do you anticipate these will translate into real-world functional outcomes as more longitudinal data emerge from the pivotal phase?

RGX-202 is unique in encoding the C-Terminal (CT) domain of dystrophin, which has been shown in preclinical models to protect muscle fibers. Could you elaborate on the potential advantages of including this domain, and how it might affect muscle resilience and repair in DMD patients?

The pivotal phase is enrolling now, with the primary endpoint focusing on ≥10% microdystrophin expression at Week 12. Given the current data, do you believe RGX-202 is well-positioned for accelerated approval, and what additional evidence will be most critical to support that regulatory path?