The Food and Drug Administration (FDA) has granted Orphan Drug designation to TN-201, an investigational adeno-associated virus (AAV)-based gene therapy, for genetic hypertrophic cardiomyopathy.

Genetic hypertrophic cardiomyopathy is a rare, inherited heart condition most commonly caused by mutations of the Myosin Binding Protein C3 (MYBPC3) gene. The condition is characterized by thickening of the heart walls, leading to abnormal heart rhythms, cardiac dysfunction, heart failure and sudden cardiac death. 

TN-201 is a potentially curative, one-time, AAV-based gene therapy to address genetic hypertrophic cardiomyopathy caused by MYBPC3 gene mutations. In preclinical studies, TN-201 showed significant reversal of left ventricular hypertrophy, cardiac dysfunction, and electrophysiological deficits in a severe murine model, which was dose-dependent and maintained for more than 1 year.

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“We are pleased that the FDA has granted orphan drug designation for TN-201,” said Faraz Ali, CEO of Tenaya. “The feedback we have received from multiple regulatory agencies supports our IND-enabling studies, planned scale-up of AAV manufacturing at our new cGMP facility, and the design of planned first-in-human clinical studies.”

The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals.


Tenaya Therapeutics receives Orphan Drug designation and presents pre-clinical data for its most advanced gene therapy product candidate for genetic hypertrophic cardiomyopathy. [press release]. South San Francisco, CA: Tenaya Therapeutics; May 20, 2021.

This article originally appeared on MPR