Advancements in Gene Therapy Programs
REGENXBIO has made significant progress in several gene therapy programs, including RGX-202 for Duchenne, RGX-121 for MPS II, and RGX-314 for wet AMD and DR. Key milestones include the initiation of BLA filing for RGX-121 and upcoming pivotal trials.
First Functional Data for Duchenne Program
The company expects to share the first functional data from the Duchenne program later this month, marking a significant step in its development.
Strong Financial Position
REGENXBIO ended the quarter with $279 million in cash, cash equivalents, and marketable securities, expected to fund operations into 2026.
Positive Data from Retinal Studies
RGX-314 for wet AMD showed a 97% reduction in treatment burden at 9 months, with 100% of patients needing 0 or 1 supplemental injections.