REGENXBIO Hunts For 2025 Gene Therapy Approval

The group’s gene therapy reduces saccharide levels in the rare childhood disease known as Hunter syndrome, but a confirmatory trial will be needed.  

hunter syndrome brain
• Source: Alamy

Regenxbio Inc. is a step closer to bringing its mucopolysaccharidosis type II (MPS II) gene therapy to market following a pivotal win in the CAMPSIITE trial.

Key Takeaways

  • REGENXBIO’s mucopolysaccharidosis type II gene therapy RGX-121 succeeded in Phase III

But the study only assessed the effect of RGX-121 on a surrogate endpoint – levels of a disaccharide called D2S6...

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