Key points from article :
Tenaya Therapeutics has raised $52.5 million through a public offering to advance its gene therapy programs for heart disease. The company is focused on developing treatments for genetic heart conditions, including hypertrophic cardiomyopathy (HCM) and arrhythmogenic right ventricular cardiomyopathy (ARVC). Its leading candidates, TN-201 and TN-401, use adeno-associated virus (AAV9) delivery to introduce functional genes into heart cells, aiming to restore protein levels and slow or reverse disease progression.
TN-201 targets MYBPC3-associated HCM, while TN-401 is designed for PKP2-associated ARVC. Early clinical trials have shown promising safety and biomarker improvements, with more data expected in 2025. The company is expanding its clinical trials internationally and expects key safety and efficacy results from both programs throughout the year.
Tenaya also received an $8 million grant from the California Institute for Regenerative Medicine (CIRM) to support the Phase 1b trial of TN-401. With continued progress in its gene therapy pipeline, the company aims to address the root causes of inherited heart disease and improve patient outcomes.