Regenxbio is recruiting for its Phase I/II Affinity Duchenne trial of the RGX-202 gene therapy treatment for Duchenne Muscular Dystrophy.

Regenxbio announced that the Phase I/II Affinity Duchenne trial of RGX-202 for the treatment of Duchenne muscular dystrophy (Duchenne) is now active and recruiting patients.

RGX-202 delivers a transgene for a novel microdystrophin protein that includes the functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin. RGX-202 uses Regenxbio’s proprietary NAV AAV8 vector.

Presence of the CT domain has been shown in preclinical studies to recruit several key proteins to the muscle cell membrane, leading to improved muscle resistance to contraction-induced muscle damage in dystrophic mice.

Additional design features, including codon optimization and reduction of CpG content, may potentially improve gene expression, increase translational efficiency and reduce immunogenicity.

“Duchenne muscular dystrophy is a devastating disease and there are still unmet therapeutic needs,” said Aravindhan Veerapandiyan, MD, a principal investigator in the study and director of the Comprehensive Neuromuscular Program, PPMD Certified Duchenne Care Center, and co-director of the Muscular Dystrophy Association Care Center at Arkansas Children’s Hospital. “Gene therapies, like RGX-202, have the potential to impact the progressive nature of Duchenne.”

Affinity Duchenne is a multicenter, open-label dose evaluation and dose expansion clinical trial to evaluate the safety, tolerability and clinical efficacy of a one-time intravenous dose of RGX-202 in patients with Duchenne.

Regenxbio is currently recruiting patients for the Affinity Beyond trial, an observational screening study. The primary objective is to evaluate the prevalence of AAV8 antibodies in patients with Duchenne up to 12 years of age. Information collected in this study may be used to identify potential participants for the Affinity Duchenne trial and potential future trials of RGX-202.

Regenxbio has manufactured additional clinical supplies of RGX-202 in its in-house manufacturing innovation center using the NAVXpress process platform. The manufacturing innovation center is designed to meet global clinical and commercial regulatory standards, and includes two independent bulk drug substance production suites, a final drug product suite and integrated quality control labs.

“I am pleased that we are now able to initiate the trial for RGX-202 and also begin enrollment activities in our AAV8 antibody screening study,” said Kenneth T. Mills, president and chief executive officer of Regenxbio. “The RGX-202 program is a key piece of our ‘5x’25’ strategy to have five AAV therapeutics either on the market or in late-stage development by 2025.”

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