REGENXBIO ANNOUNCES NEW POSITIVE DATA FROM AFFINITY DUCHENNE® TRIAL OF RGX-202

Link to press release

We are thrilled to share the latest data from RegenXBio’s microdystrophin gene therapy,  AFFINITY DUCHENNE®. RGX-202 is a one-time gene therapy for Duchenne designed to deliver a novel microdystrophin gene via AAV8.  RGX-202 is differentiated from other microdystrophin gene therapies in that it contains a larger portion of the C-Terminal domain, which RegenXBio hopes will better protect muscle from stress. 

Duchenne Gene Therapy

RGX-202 has been well tolerated with no serious adverse events.  In this latest data, two additional individuals treated with the pivotal dose demonstrated robust microdystrophin expression: individuals aged 5.8 and 8.5 years showed expression levels at 77.2% and 46.5% of control, respectively.   RegenXBio stated that expression levels across all 7 individuals treated continues to support plans for accelerated approval, showing meaningful increases in microdystrophin expression and reductions in creatine kinase levels at their 3-month assessments. 

REGENXBIO expects to complete enrollment in this dose level 2 expansion cohort early in Q3 2024, and has initiated enrollment for boys aged 1-3.  Initiation of their pivotal trial is expected in Q4 2024.