RGX-202-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-202 in participants who have received RGX-202 (an investigative gene therapy designed to deliver a transgene for novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain) in a separate parent study.
Current status: ENROLLING BY INVITATION.
REGENXBIO Inc (RGNX) is the sponsoring company for this trial. BiotechSign currently grades this company D (42/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.