Charlie [Analyst]'s questions to Regenxbio Inc (RGNX) leadership • Q3 2024
Question
Charlie [Analyst], on for Brian Skorney at Baird, asked about the challenges in identifying Duchenne patients in the 1-3 year old cohort, given the typical diagnosis age is 4-5, and what efforts could be made to lower the average age of diagnosis.
Answer
Dr. Steve Pakola, CMO, acknowledged the traditional diagnosis age but noted that the increasing adoption of newborn screening is helping to identify patients earlier. He confirmed they have already dosed the first patient in this cohort, indicating they are able to find these patients. He believes the compelling profile of RGX-202 will attract families once a diagnosis is made.