REGENXBIO Publishes Preclinical Data Highlighting RGX-202’s Potential for Duchenne Muscular Dystrophy

REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO announced the publication of preclinical results for its investigational gene therapy, called RGX-202, for Duchenne Muscular Dystrophy. The findings, published in the peer-reviewed journal Molecular Therapy Methods and Clinical Development, showed that a microdystrophin gene therapy construct including the C-terminal/CT domain showed improved functional benefits compared to a construct without it.

RGX-202 is unique among investigational or approved microdystrophin gene therapies for Duchenne in that it includes this crucial CT domain, making it the closest to naturally occurring dystrophin. The preclinical study evaluated two AAV vectors in mdx mice, a model for Duchenne. One vector encoded a microdystrophin protein with the CT domain, while the other encoded an equivalent microdystrophin without it.

A medical professional holding a branded prescription drug manufactured by the pharmaceutical company.

The results showed that the microdystrophin with the CT domain was maintained at higher levels in transduced muscles, more effectively recruited the dystrophin-associated protein complex to the muscle membrane, and led to increased muscle force and resistance to damage in the dystrophin-deficient mice. These findings suggest that the CT domain enhances the microdystrophin design by promoting higher protein accumulation due to a longer half-life, which can lead to improved functional benefits.

REGENXBIO Inc. (NASDAQ:RGNX) is a clinical-stage biotechnology company that provides gene therapies that deliver functional genes to cells with genetic defects in the US.

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Disclosure: None. This article is originally published at Insider Monkey.