Regenxbio (RGNX) 2026 Global Healthcare Conference summary

Strategic focus and platform overview

• Fifteen years of expertise in AAV8 and AAV9 gene therapy, with programs in retina and rare diseases.

• Advanced manufacturing enables high-dose AAV with 80% full capsid purity, appealing to regulators.

• Institutional knowledge leveraged across programs for safety and efficacy.

Duchenne muscular dystrophy (DMD) program (RGX-202)

• RGX-202 features a unique C-terminus in microdystrophin, aiming for muscle repair and high functional benefit.

• Lower SAE rates and high, consistent microdystrophin expression observed, with strong functional improvements.

• Top-line data supports BLA submission, targeted for Q1 next year, with 63 patients treated to date.

• Strong correlation (0.9) between microdystrophin expression and functional benefit, surpassing competitors.

• Confirmatory AFFINITY study fully enrolled, designed for broad patient inclusion and long-term safety follow-up.

Regulatory and global strategy

• BLA for Hunter syndrome submitted; RGX-202 BLA non-clinical section complete, with pre-BLA meeting planned.

• Active engagement with FDA leadership, aiming for accelerated approval due to high unmet need.

• European approval strategy includes a randomized controlled trial (RCT) actively enrolling during U.S. review.