REGENXBIO has concluded enrolment of subjects in the AFFINITY DUCHENNE clinical study evaluating its RGX-202 gene therapy candidate for Duchenne muscular dystrophy (DMD).
The pivotal stage of the open-label, multi-centre, Phase I/II/III trial enrolled 30 subjects. The study’s main goal is the proportion of subjects achieving RGX-202 microdystrophin expression of 10% or more at week 12, supporting accelerated approval.
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Secondary assessments include baseline changes in timed functional tests among subjects aged four years and above, while those aged one to less than four years will be assessed with the SV95C and peabody developmental motor scale-third edition (PDMS-3).
In the trial’s Phase I/II segment, microdystrophin concentration ranged from 20 to 122% after the pivotal dose.
As of May 2025, RGX-202 displayed favourable safety, with no serious adverse events (SAEs) and adverse events of special interest (AESIs) reported.
REGENXBIO president and CEO Curran Simpson said: “The Duchenne community urgently needs new treatment options that provide durable, safe outcomes and can meaningfully change the course of this degenerative disease.
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By GlobalData“Completing this pivotal trial milestone and manufacturing in-house our first doses intended for commercial use bring us even closer to delivering RGX-202 as a potential best-in-class gene therapy for Duchenne patients with limited options.
“The differentiated therapeutic approach behind RGX-202, including our industry-leading product purity levels and novel construct with the C-Terminal domain, has resulted in the positive safety and efficacy profile, with consistent functional benefit seen in Phase I/II.”
The company expects to share top line pivotal data in the second quarter of next year.
REGENXBIO has also started production of the initial RGX-202 batches for supply commercially and produced a full supply for the confirmatory study, anticipating a 2027 market launch.
The therapy is manufactured at the company’s innovation centre in Rockville, Maryland, US, using the NAVXpress suspension-based process, yielding more than 80% full capsids. The plant’s annual capacity is 2,500 doses.
Earlier this month, REGENXBIO concluded participant enrolment in the pivotal ATMOSPHERE and ASCENT trials, which assessed surabgene lomparvovec (sura-vec, ABBV-RGX-314) for the treatment of wet age-related macular degeneration (wet AMD) via subretinal delivery.
Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.
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