The therapy's favorable safety profile is likely due to a proactive immune modulation regimen and high product purity levels.

Safety data indicated no serious adverse events, with only mild effects like nausea and fatigue reported, which are common in gene therapy.

The primary endpoint of the pivotal phase of the AFFINITY DUCHENNE study is to achieve at least 10% microdystrophin expression by week twelve; early results have shown high expression levels, including one patient reaching 118.6%.

An interim analysis involving the first five participants from the dose level 2 cohort revealed consistent benefits at both nine and twelve months post-treatment.

Enrollment is ongoing for the phase 3 segment of the AFFINITY DUCHENNE trial, which aims to include approximately 30 participants.

Changes in timed task velocity exceeded clinically significant benchmarks at the twelve-month evaluation for the RGX-202 group.

Despite expectations that four out of five participants would experience a decline in their disease trajectory, those treated with RGX-202 showed improved scores in both the NSAA and timed function tests.

RGX-202, an investigational gene therapy for Duchenne muscular dystrophy (DMD), has demonstrated significant functional improvements in participants compared to natural history controls during the AFFINITY DUCHENNE trial.

At the nine-month mark, treated patients showed an average increase of 4 points in their North Star Ambulatory Assessment (NSAA) scores from baseline, and a 4.8-point improvement compared to controls, with similar results observed at twelve months.

Experts are optimistic about RGX-202's potential to delay disease progression in DMD, highlighting the critical need for innovative therapies aimed at muscle preservation.