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Tenaya Therapeutics receives orphan drug designation for TN-401

Tenaya Therapeutics announced that the U.S. Food and Drug Administration has granted orphan drug designation for its second gene therapy product candidate, TN-401, for the treatment of arrhythmogenic right ventricular cardiomyopathy. TN-401 is an adeno-associated virus-based gene therapy being developed for the treatment of genetic ARVC caused by Plakophilin-2 gene mutations. Mutations of the PKP2 gene can cause severe disease, including enlargement of the right ventricle in affected individuals, cardiac dysfunction, significant arrhythmia and sudden cardiac death in adults and children. PKP2 mutations are the most common genetic cause of ARVC, estimated to represent approximately 40 percent of the overall ARVC population and to effect more than 70,000 people in the U.S. alone. TN-401 is designed to use an AAV9 vector to deliver a healthy copy of the PKP2 gene to the heart muscle of affected patients via a single intravenous dose. Current treatments do not address the underlying genetic cause of disease and do not appear to affect disease progression. Tenaya intends to initiate a global non-interventional study of ARVC PKP2 gene mutation carriers by the end of this year in order to collect treatment history and data on seroprevalence to AAV antibodies. Tenaya expects to submit an Investigational New Drug application for the program to the FDA in 2023.

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