Regenxbio presented interim Affinity trial data showing functional improvements and a clean safety profile for its DMD gene therapy candidate RGX‑202, the company said at the Muscular Dystrophy Association conference. The phase I/II data show durable functional signals and stable cardiac metrics in treated patients, reinforcing confidence ahead of an upcoming pivotal dataset. Company commentary framed results as supportive of continued development while investors and regulators watch for pivotal endpoints. The findings arrive amid heightened regulatory scrutiny in the DMD space and underscore the ongoing debate over risk–benefit calculations for systemic gene therapies in pediatric rare disease.