Tenaya Therapeutics announced the publication of its preclinical research related to its gene therapy candidate, TN-401, in the current issue of Nature Communications Medicine. “Following a single infusion of our AAV9-based PKP2 gene therapy in a severe knock-out mouse model of the disease, PKP2 protein levels were restored. This led to dose-dependent improvements in right ventricular dilation and ejection fraction, reductions in arrhythmia frequency and severity, and prevention of adverse fibrotic remodeling, with near-maximal efficacy achieved at the 3E13 vg/kg dose.” said Tim Hoey, Ph.D., Chief Scientific Officer of Tenaya.
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