Regenxbio (RGNX) 2026 Global Healthcare Conference summary

Company overview and core competencies

• Focuses on AAV gene therapy with expertise in AAV8 and AAV9 capsids, targeting retina and rare diseases.

• Leverages 15 years of institutional knowledge to advance clinical programs.

• Leads in safe, high-dose AAV delivery and manufacturing, achieving 80% full capsid purity.

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

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

• Demonstrates lower SAE rates and higher, more consistent microdystrophin expression than competitors.

• Shows strong correlation (0.9) between microdystrophin levels and functional benefit (NSAA), supporting efficacy.

• Top-line data supports BLA submission, targeted for Q1 next year, with pre-BLA meeting planned.

• Confirmatory and pivotal studies fully enrolled, with broad patient inclusion and robust safety follow-up.

Regulatory and clinical development

• BLA for Hunter syndrome recently submitted, providing organizational experience for RGX-202 BLA.

• FDA leadership engaged, supportive of accelerated approval due to unmet need in DMD.

• European approval strategy includes an actively enrolling RCT for RGX-202.