Clinical Trial SuccessThe RGX-202 pivotal trial in Duchenne muscular dystrophy (DMD) has completed enrollment and reported promising results with no serious adverse events, indicating strong potential for future approval and market success.
Manufacturing CapabilitiesRegenxbio's end-to-end capabilities and ability to safely provide doses at commercial scale with attractive cost of goods sold (COGS) is seen as a strength versus competition.
Regulatory ApprovalsRGX-121 has received multiple designations from the FDA, including Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT), which could expedite regulatory processes and increase market potential.