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ROCKVILLE, Md. - REGENXBIO Inc. (NASDAQ:RGNX), a biotechnology company whose stock has gained over 22% in the past six months and is currently considered undervalued according to InvestingPro analysis, published preclinical research Thursday showing that its microdystrophin gene therapy construct including the C-terminal (CT) domain demonstrated superior results compared to versions without this domain in treating Duchenne muscular dystrophy.
The study, published in Molecular Therapy Methods and Clinical Development, found that the microdystrophin with CT domain maintained higher protein levels in muscles, increased muscle force, and improved resistance to damage in mice lacking dystrophin. With revenue growth of 80.7% over the last twelve months and a strong balance sheet showing more cash than debt, REGENXBIO appears well-positioned to advance its research programs.
According to the company, RGX-202 is the only investigational or approved microdystrophin gene therapy for Duchenne that includes the CT domain, making it closer to naturally occurring dystrophin.
"The microdystrophin that included the CT domain was found to be maintained at higher levels in transduced muscles, recruited the dystrophin-associated protein complex more effectively to the muscle membrane, and increased muscle force and resistance to damage," the study reported.
The company stated these findings support interim results from its ongoing Phase I/II AFFINITY DUCHENNE clinical trial, which in June showed evidence of positive changes in disease trajectory with a favorable safety profile.
Duchenne muscular dystrophy affects 1 in 3,500 to 5,000 boys born worldwide and causes progressive muscle degeneration due to mutations in the dystrophin gene.
REGENXBIO is currently enrolling participants in the pivotal portion of its Phase I/II/III trial and expects to submit a Biologics License Application using the accelerated approval pathway in mid-2026.
The company manufactures RGX-202 using its proprietary NAVXpress suspension-based platform process. The information in this article is based on a company press release. For deeper insights into REGENXBIO’s financial health and growth prospects, including 8 additional ProTips and comprehensive analysis, visit InvestingPro, where you’ll find detailed research reports covering 1,400+ top stocks.
In other recent news, Regenxbio Inc. reported positive interim data from its Phase I/II AFFINITY DUCHENNE trial, which showed improvements in patients treated with RGX-202 for Duchenne muscular dystrophy. The data indicated significant functional gains and a slowdown in disease progression, with favorable safety profiles and high microdystrophin expression levels. Analysts from H.C. Wainwright and Stifel have both maintained their Buy ratings on Regenxbio, with price targets set at $34 and $40, respectively, reflecting confidence in the company’s potential. Additionally, Regenxbio has secured a financial agreement with Healthcare Royalty, receiving $150 million upfront, potentially extending to $250 million, to support its ongoing and future projects without diluting shareholder equity. This deal aims to bolster Regenxbio’s cash runway into early 2027 and facilitate the development of its gene therapy pipeline. The company plans to submit a Biologics License Application for RGX-202 by mid-2026, with pivotal trial data expected in the first half of that year. Regenxbio’s strategic financial positioning is further strengthened by anticipated non-dilutive funding sources, including milestones from its collaboration with AbbVie. These developments underscore Regenxbio’s commitment to advancing its treatments for rare diseases.
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