Regenxbio’s Triple Threat: Partnered Programs, DMD Progress And In-House Manufacturing

Regenxbio is riding a wave these days, with all three of its clinical-stage programs set to reach critical milestones this year and recent positive news on its DMD gene therapy, just as Sarepta suffered a setback with a death related to its DMD treatment. In Vivo spoke with CEO Curran Simpson about the company’s plans and strategic decisions.

Regenxbio
• Source: Alamy

It’s a big year for Regenxbio. The company has a potential commercial launch of its gene therapy program for MPS II (Hunter syndrome), a Biologics License Application for its Duchenne muscular dystrophy (DMD) gene therapy, and topline data from a pivotal study of its AbbVie-partnered wet age-related macular degeneration (AMD) gene therapy.

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