Regenxbio has reported positive interim data from the Phase I/II portion of its Affinity Duchenne study (NCT05693142), which is examining its investigational gene therapy RGX-202. The therapy is ...

Positive biomarker data in patient aged 1-3 add to consistent, robust microdystrophin and transduction levels across all treated agesPatient aged 3 years at dosing had expression level at 122.3% ...

Regenxbio plans to submit a BLA for RGX-202 in mid-2026, with Phase 3 enrollment ongoing. In a market dominated by tariff tensions, geopolitical surprises, and Fed uncertainty, Matt Maley's ...

"RGX-202 is the only next generation gene therapy for Duchenne in a pivotal phase trial. The new data from the age 1-3 cohort builds on the favorable safety and efficacy profile seen in ages 4 and ...