Gene TherapyDeveloping
FDA Reopened a Path for a One-Time Hunter Syndrome Gene Therapy
Regenxbio says FDA no longer requires additional studies before reviewing Navsunli/RGX-121 for accelerated approval, a potentially important rare-disease regulatory signal.
Why now
FDA has reopened a filing path after the program’s regulatory outlook had narrowed.
- Overclaim risk
- high
- Primary source
- Company
- Published
- Jun 24, 2026
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