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Varfaj, Fatbardha

Mayo Clinic

Tenaya Therapeutics , Inc., South San Francisco, CA, USA

Citations 1

First-in-human study of TN-201, an AAV9 gene replacement therapy in MYBPC3 -associated hypertrophic cardiomyopathy: initial safety, pharmacodynamic, and imaging results from MyPEAK-1
Desai, Milind Y. , Nagueh, Sherif F. , Giudicessi, John R. , Kellner, Danielle, Pollman, Matthew J., Varfaj, Fatbardha, Robertson, Laura, Sonicheva-Paterson, Natasha, Pushkin, Richard, Mangal, Brian, Tomlinson, LaTanya, Harrison, W. G., Yamamoto, Lynn, Argast, Gretchen, Ivey, Kathryn N. , Tingley, Whittemore G., Previs, Michael J. , Sonicheva‐Paterson, Natalia , Harrison, William G, Yamamoto, Lynn, Lombardi, Laura Marie

Cardiovascular Research | November 9, 2025 | Volume: 121 | Issue: 17 | Pages: 2628-2631

10.1093/cvr/cvaf200 Get It

Abstract This brief report details the initial findings from a Phase 1b/2 trial of TN-201, an adeno-associated virus serotype 9 (AAV9) gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), a condition with significant morbidity, increased risk of mortality, and no approved therapy for the majority of patients. TN-201 was well tolerated, and changes to the management of potential immune responses resulted in a shorter period of immunosuppression. These results show consistent transduction and expression of TN-201 in cardiomyocytes, corresponding with increases in MyBP-C levels, reductions or stabilization of cardiac biomarkers, and reductions in key measures of left ventricular (LV) hypertrophy.