Tenaya Therapeutics (TNYA) presented preclinical data evaluating TN-301, the company’s highly selective HDAC6 inhibitor, at the Muscular Dystrophy Association’s Clinical & Scientific Conference 2026. In in vitro and in vivo models of Duchenne muscular dystrophy, TN-301 improved muscle performance and corrected key drivers of DMD cardiomyopathy. Key findings presented at MDA 2026 include: TN-301 treatment at doses as low as 3 mg/kg improved grip strength to wild-type levels within five weeks, whereas mdx mice treated with givinostat failed to reach wild-type performance. TN-301-mediated functional improvements were accompanied by reductions in circulating creatine kinase activity and favorable changes in gene expression, indicating reduced muscle cell injury. In cardiomyocytes derived from human DMD induced pluripotent stem cells, TN-301 corrected calcium handling abnormalities and mitochondrial dysfunction, while givinostat exacerbated these established drivers of DMD cardiomyopathy.
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