Summary of Tenaya Therapeutics (TNYA) FY Conference Call Company Overview - **Company**: Tenaya Therapeutics (TNYA) - **Founded**: 2016 - **Mission**: Focus on therapies for heart disease, including both rare genetic forms and prevalent forms [3][4] Pipeline and Clinical Development - **Clinical Stage Assets**: Three clinical stage assets currently in development [4] - **Gene Therapies**: Focus on gene therapies for genetic causes of cardiomyopathy and a small molecule for HFpEF [4] - **Clinical Sites**: Over 40 clinical sites active in seven countries for patient recruitment [4] Gene Therapy Programs TN-201 for MYBPC3 Positive Hypertrophic Cardiomyopathy - **Target Disease**: Leading genetic cause of hypertrophic cardiomyopathy, affecting approximately 120,000 patients in the US [15] - **Mechanism**: Addresses deficiency of myBPC3 protein, leading to heart thickening and potential heart failure [16][17] - **Natural History Study**: MyCLIMB study with over 220 children enrolled to characterize disease progression [20][21] - **Phase 1b Study**: Completed dosing of high dose cohort; initial results show improvement in heart function [25][27] - **Upcoming Data**: Full cohort data expected in Q4 2025, with focus on safety and efficacy [37] TN-401 for PKP2-Related Arrhythmogenic Right Ventricular Cardiomyopathy - **Target Disease**: Accounts for about 40% of arrhythmogenic right ventricular cardiomyopathy cases, approximately 70,000 patients in the US [44] - **Mechanism**: Aims to add a copy of the human gene to improve desmosome function and reduce arrhythmia burden [46] - **Biopsy Data**: Expected in Q4 2025, focusing on vector copy number, RNA, and protein levels [50][51] Safety and Efficacy - **AAV9 Vector**: Selected for its extensive safety database and effectiveness in cardiac applications [5][6] - **Safety Record**: No significant safety issues reported in ongoing trials; adverse events consistent with other gene therapies [13][14] - **Patient Outcomes**: Initial data shows symptomatic improvement in patients treated with TN-201 [25][27] Small Molecule Program TN-301 - **Status**: Completed first-in-human study with 72 patients; well tolerated with no dose-limiting toxicities [55] - **Future Plans**: Exploring opportunities to advance the program, focusing on HFpEF and potentially severe rare diseases [56] Key Takeaways - **Market Opportunity**: Both TN-201 and TN-401 target large orphan conditions with significant unmet medical needs [44][46] - **Data Releases**: Important data updates expected in Q4 2025 for both gene therapy programs, which could influence future clinical development strategies [37][50]