TN-201

TN-201 is an investigational gene therapy developed by Tenaya Therapeutics for the treatment of hypertrophic cardiomyopathy (HCM) caused by mutations in the MYBPC3 gene. It utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the MYBPC3 gene specifically to heart muscle cells via a cardiomyocyte-specific promoter. The goal is to restore or increase levels of myosin-binding protein C (MyBP-C), thereby addressing the underlying genetic cause of disease, slowing progression, and potentially reversing cardiac dysfunction. TN-201 is administered as a one-time intravenous infusion and has received orphan drug status, rare pediatric disease designation, and fast track designation from regulatory agencies for HCM. Early clinical data show that it is generally well tolerated with mild or manageable adverse events typical of AAV-based therapies[1][2][4][5][6][8][10].