Qian Wang's questions to Regenxbio Inc (RGNX) leadership • Q1 2025
Question
Qian Wang from Barclays asked about potential FDA safety requirement changes for DMD gene therapies following a competitor's safety event, the impact on RGX-202's trial enrollment, and whether the new CBER Director might alter the accelerated approval path.
Answer
President and CEO Curran Simpson saw no signs of change to the accelerated approval path and noted their enrollment is on track. He highlighted RGX-202's differentiated safety profile and innovative immune suppression regimen. Chief Medical Officer Dr. Steve Pakola added that the competitor event brightens the spotlight on safety, which reinforces the value of their differentiated approach and gives the community comfort.