The AFFINITY DUCHENNE trial reported microdystrophin expression levels of 77.2% in a 5.8-year-old patient and 46.5% in an 8.5-year-old patient at dose level 2. How do these results compare with existing gene therapies, and what implications do they have for younger versus older patients with Duchenne?

Given the robust microdystrophin expression observed across all treated patients, particularly at higher dose levels, how do you foresee RGX-202 impacting the progression of Duchenne? Could this potentially alter the standard of care for these patients?

RGX-202 has been reported as well-tolerated with no serious adverse events in the trial. What are the key factors contributing to this safety profile, and how does it compare to other gene therapies currently in development for Duchenne?

With the consistent, high expression of microdystrophin and reduction in serum creatinine kinase levels, REGENXBIO is planning for accelerated approval. What are the critical steps needed for this to happen, and what might the timeline look like?

REGENXBIO has initiated enrollment for boys aged 1-3 in the trial. What challenges and opportunities do you see in treating this younger population, and how might early intervention impact long-term outcomes?

RGX-202 is unique in encoding the C-Terminal domain of dystrophin, which is suggested to enhance muscle repair and protection. Could you elaborate on the importance of this domain and how it might differentiate RGX-202 from other gene therapies?

REGENXBIO expects to share initial strength and functional assessment data later this year. What specific functional improvements would you consider most indicative of RGX-202’s effectiveness in Duchenne treatment?

What aspects of clinical trial design might allow REGENXBIO to pursue an accelerated approval strategy? 

What is the current thinking around the presence or absence of the hinge 2 domain in the transgene? RGX-202 does not contain hinge 2, and Pfizer's '926 didn't either. Elevidys does contain hinge 2. (Banks et al. 2010 found that the polyproline site in hinge 2 profoundly affects the functional capacities of microdystrophin) 

Why did the interviewer spend over 20 min discussing Elevidys and myostatin inhibitors on a call that was supposed focus on RGX-202??