Tenaya Therapeutics(US:TNYA)2025-08-13 20:00Pipeline and Programs - Tenaya Therapeutics has 3 clinical-stage programs focused on heart disease[9] - TN-201, a gene therapy for MYBPC3-associated HCM, is in Phase 1b/2 clinical trials with data readouts expected in Q4 2025[10, 12] - TN-401, a gene therapy for PKP2-associated ARVC, is in Phase 1b clinical trials with initial data including safety and biopsy results expected in 2025[10, 94] - TN-301, a small molecule HDAC6 inhibitor for HFpEF, has completed Phase 1 trials and is seeking a partner for further development[10, 99, 104] TN-201 for MYBPC3-associated HCM - MYBPC3-associated HCM affects an estimated 120,000 people in the U S alone[22] - Interim Cohort 1 data for TN-201 showed the therapy was well-tolerated at 3E13 vg/kg dose, with no cardiotoxicities and manageable liver enzyme elevations[17, 40] - All three patients in Cohort 1 improved from NYHA class II/III to NYHA class I, and elevated troponin levels dropped by 60% in two patients into normal ranges[19] - TN-201 DNA in cardiac biopsy surpassed preclinical efficacy threshold and compares favorably to peer, with long-term stability achieved[42] - TN-201 RNA expression increased by as much as 13-fold from week 8 to Week 52[46] TN-401 for PKP2-associated ARVC - PKP2-associated ARVC is estimated to affect over 70,000 people in the U S [75] - Preclinical studies showed that a single 3E13 vg/kg dose of TN-401 in a KO mouse model reversed hallmarks of the disease and extended survival[91] - RIDGE natural history study shows that over 80% of ARVC patients continue to experience high PVC count despite standard of care treatments[87]