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REGENXBIO Announces Publication of Preclinical Results Demonstrating Functional Benefits of Novel Microdystrophin Construct in RGX-202 Investigational Gene Therapy for Duchenne Muscular Dystrophy
Construct including CT domain demonstrated higher levels of microdystrophin protein, increased muscle force, and improved resistance to damage in mice lacking
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[{"type":"text","content":"Construct including CT domain demonstrated higher levels of microdystrophin protein, increased muscle force, and improved resistance to damage in mice lacking dystrophin REGENXBIO's next-generation investigational gene therapy, RGX-202, is the only microdystrophin construct that includes the CT domain Findings support the positive functional data seen in Phase I/II AFFINITY DUCHENNE® trial of RGX-202ROCKVILLE, Md., July 10, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the publication of preclinical results comparing a microdystrophin gene therapy construct that included the C-terminal (CT) domain to a microdystrophin construct without the CT domain. The results, which were published in peer-reviewed journal Molecular Therapy Methods and Clinical Development, showed that the microdystrophin with the CT domain improved functional benefit compared to the microdystrophin without, supporting the potential of RGX-202 to drive functional improvements in patients with Duchenne Muscular Dystrophy.\nRGX-202 is the only investigational or approved microdystrophin gene therapy candidate for the treatment of Duchenne muscular dystrophy (Duchenne) that includes the CT domain, a key portion of dystrophin, making it the closest to naturally occurring dystrophin.\n\"We specifically designed RGX-202 differently from other gene therapies with the goal of providing improved outcomes for patients, and this research further validates the potential therapeutic advantage of adding the CT domain and its importance in preventing the muscle breakdown associated with functional decline in Duchenne,\" said Olivier Danos, Ph.D., Chief Scientific Officer of REGENXBIO. \"The positive interim results we've seen in the Phase I/II AFFINITY DUCHENNE® trial are reinforced by this preclinical research demonstrating how the novel construct of RGX-202 protects against muscle damage and supports the potential for durable, functional benefit for patients.\"\n\"AAV gene therapy holds great promise for Duchenne, and the community is in need of treatment options that have the potential to improve function and quality of life for patients,\" said Michael Kelly, Ph.D., Chief Scientific Officer, CureDuchenne. \"The CT domain is a critical part of the large DMD gene and these preclinical results highlight its role in muscle health. With the science behind...