REGENXBIO Inc (NASDAQ:RGNX) reported additional interim safety and efficacy data in the Phase 1/2 AFFINITY DUCHENNE trial of RGX-202 in patients with Duchenne muscular dystrophy (Duchenne) ages 4 to11 years old.
As of February 28, 2024, RGX-202 has been well tolerated with no drug-related serious adverse events in five patients aged 4.4 to 12.1 at dose level 1 (1×1014 genome copies (GC)/kg body weight) and dose level 2 (2×1014 GC/kg body weight).
In new data from the first patient, aged 12.1 years, who received RGX-202 at dose level 2, RGX-202 microdystrophin expression was measured to be 75.7% ...