Regenxbio has presented biomarker data from its Duchenne muscular dystrophy gene therapy trial, demonstrating near-normal microdystrophin expression in a 3-year-old patient.
The company reported at the Muscular Dystrophy Association’s annual scientific conference that a 3-year-old patient treated with its gene therapy, RGX-202, showed microdystrophin expression at 122.3% of healthy control levels. This data supports Regenxbio’s ongoing trial in children aged 1 to 11, aiming for approval in younger patients.
Regenxbio also shared updated biomarker results from older children. Two patients aged 4 to 7 showed microdystrophin expression of 54.3% of normal, while five patients aged 8 to 11 showed 39.7% of normal.
“It’s not surprising that younger patients express more protein, consistent with observations from other microdystrophin gene therapies,” said Regenxbio CMO Steve Pakola. He noted that Duchenne’s progressive nature may lead to reduced expression in older patients due to muscle scarring, but emphasized the decrease was not substantial.
Microdystrophin expression ranges were similar across older age groups: 31.5% and 77.2% in the younger group, and 20.8% to 75.7% in the older group.
Regenxbio plans to seek FDA accelerated approval for RGX-202 by mid-2026, using a three-month microdystrophin endpoint. The pivotal Phase 1/2/3 study, enrolling patients aged 1 and older, is approximately halfway through dosing its 30-patient cohort. Topline results are expected in the first half of 2026.
Regenxbio aims for a broad label, seeking to expand access beyond the current age restrictions seen in Sarepta Therapeutics’ Elevidys, which is approved for patients aged 4 and older.
Source:
https://endpts.com/regenxbio-shares-new-duchenne-gene-therapy-data-ahead-of-big-readout-next-year/
About PackGene
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
