Regenxbio expands Phase I/II AFFINITY DUCHENNE trial

Regenxbio announced it initiated enrollment in a new cohort of patients ages 1-3 in its Phase I/II AFFINITY DUCHENNE trial to evaluate the safety and efficacy of RGX-202 in boys with Duchenne muscular dystrophy. RGX-202 is an investigational one-time AAV Therapeutic targeted to deliver a novel microdystrophin, representing the next wave of innovative design in Duchenne gene therapy. RGX-202 is the only gene therapy approved or in development for Duchenne that incorporates the C-Terminal domain, making the RGX-202 transgene the closest to the naturally occurring dystrophin gene. With this announcement, REGENXBIO is enrolling ambulatory boys with Duchenne aged 1 to 11 in the AFFINITY DUCHENNE trial. The new cohort is expected to enroll up to five patients aged 1-3 to receive RGX-202 at the pivotal dose level (2×1014 genome copies.