REGENXBIO has announced it initiated enrollment in a new cohort of patients ages 1-3 in its Phase I/II AFFINITY DUCHENNE® trial. The trial is evaluating the safety and efficacy of RGX-202, an investigational gene therapy that utilizes a novel adeno-associated virus (AAV8) to transport a shortened version of the dystrophin gene (micro-dystrophin) that may provide benefit in place of the missing full-length dystrophin protein.

REGENXBIO is now enrolling ambulatory patients with Duchenne aged 1 to 11 in the AFFINITY DUCHENNE trial. According to the company, the new cohort is expected to enroll up to five patients aged 1-3 to receive RGX-202 at the pivotal dose level (2×10^14 genome copies (GC)/kg body weight).

Additionally, REGENXBIO has confirmed an end-of-Phase II meeting is scheduled with the FDA at the end of July. REGENXBIO reports that this meeting is expected to finalize the AFFINITY DUCHENNE pivotal trial design, with the goal of continuing to expedite the development of RGX-202. 

According to REGENXBIO, the company expects to share initial strength and functional assessment data for both dose levels of the AFFINITY DUCHENNE trial in the second half of 2024. Additionally, initiation of the pivotal trial is on track for late third quarter to early fourth quarter of 2024.

PPMD is pleased to learn this news and looks forward to further updates from REGENXBIO.

REGENXBIO will be participating in PPMD’s 30th Annual Conference in Orlando, Florida during the “Research Row: Gene Therapy – Current and Evolving Landscape” session on Friday, June 28. Learn more about the Conference and how to register to attend virtually here.

Read REGENXBIO’s press release here.