Tenaya Therapeutics is a clinical-stage biotech focused exclusively on developing disease-modifying gene therapies and small molecule drugs for heart disease. Tenaya has three lead programs. TN-201 is an AAV9-based gene therapy for MYBPC3-associated hypertrophic cardiomyopathy — a thickening of the heart walls caused by a genetic mutation — delivered as a single IV infusion to restore normal protein function; it is currently in a Phase 1b/2 trial. TN-401 is an AAV9-based gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy, a progressive disease causing dangerous arrhythmias and sudden cardiac death; it is also in a Phase 1b/2 trial. TN-301 is a small molecule HDAC6 inhibitor targeting inflammation, fibrosis, and metabolic dysfunction in the heart, with a primary focus on heart failure with preserved ejection fraction (HFpEF); Tenaya plans to partner TN-301 for large indications rather than develop it independently. Tenaya is pre-revenue, funding operations through equity and a research collaboration with Alnylam Pharmaceuticals to identify novel cardiovascular gene targets. For its rare disease programs, Tenaya plans to develop and commercialize independently using a focused salesforce; for larger indications like HFpEF, Tenaya plans to out-license, collecting milestones and royalties. Tenaya's platform includes iPSC-derived disease models, AAV capsid engineering, and machine learning-based screening tools to support its pipeline.
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