A potentially disease-modifying therapy for osteoarthritis (OA) has reached a key milestone now that a U.S. patent has been issued for FX201, a gene-therapy product candidate licensed by Flexion Therapeutics based on work led by Baylor College of Medicine researchers.
More than 30 million people in the United States alone are affected by OA, a degenerative joint disease, and that number is expected to rise. While currently available therapies can help manage the symptoms of OA, there are no approved treatments to address the underlying cause of the disease, which is why Dr. Brendan Lee, chair and Robert and Janice McNair Endowed Chair and Professor in the Department of Molecular and Human Genetics at Baylor, and his colleagues have been focused on finding a new way to treat this condition.
In past studies they and others have found that the interleukin-1 pathway plays a role in inflammation and is a potentially effective target in OA. To address the issue of limited availability of interleukin-1 receptor antagonist (IL-1Ra), a protein that impedes the inflammatory nature of the interleukin-1 pathway, the research team developed a helper-dependent adenovirus-mediated gene therapy to enable the long-term expression of IL-1Ra, thereby resulting in a potential sustained symptomatic and disease-modifying therapy for OA.