Tenaya Therapeutics saw a significant boost in its shares, with a 16% increase to $2.55, following the announcement that the first patient has been treated with TN-201 gene therapy for the treatment of Myosin Binding Protein C3-associated hypertrophic cardiomyopathy.
TN-201, Tenaya’s innovative adeno-associated virus-based gene therapy, aims to deliver a functioning copy of the human MYBPC3 gene to heart muscle cells. Promising preclinical studies have shown that TN-201 not only halted disease progression, but also provided substantial and long-lasting disease reversal and survival benefits after just one dose.
The Phase 1b clinical trial of TN-201 is currently underway. This dose-escalating study aims to evaluate the safety, tolerability, and clinical effectiveness of a single intravenous infusion of TN-201. The trial is expected to treat a total of up to 15 subjects from both the U.S. and international locations.
Upon dosing three patients, an external data safety and monitoring board will review the safety data and provide advice on potentially enrolling patients at a higher dose level, as well as recruiting additional patients in the initial dose cohort.
Tenaya Therapeutics is confident in sharing initial data from the Phase 1b clinical trial in 2024, marking a significant milestone in the development of this groundbreaking gene therapy.