Defeat Duchenne Canada is pleased to share the news that REGENXBIO has received clearance from the U.S. Food and Drug Administration (FDA) on their investigational gene therapy drug, RGX-2022. This means that they can move forward with the initiation of their first clinical trial with ambulatory boys aged 4-11 years with Duchenne muscular dystrophy. They hope the trial will begin in the first half of 2022.

We expect this Phase I/II trial, named AFFINITY DUCHENNE™, to open in the United States, with additional sites in Canada and Europe expected to follow. We will continue to monitor their progress and will inform you as more information becomes available.

Click the button below to read the REGENXBIO community letter:

Click the button below to read the full press release:

About RGX-202

RGX-202 is designed to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin. 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. RGX-202 is designed to support the delivery and targeted expression of genes throughout skeletal and heart muscle using the NAV AAV8 vector, a vector used in numerous clinical trials, and a well-characterized muscle-specific promoter (Spc5-12).