First-in-human study of TN-201, an AAV9 gene replacement therapy in <i>MYBPC3</i> -associated hypertrophic cardiomyopathy: initial safety, pharmacodynamic, and imaging results from MyPEAK-1

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.

Authors

Institutions

Publication Details

Journal
Cardiovascular Research
Published
2025-11-08
DOI
https://doi.org/10.1093/cvr/cvaf200
Citations
4
Primary Topic
Cardiomyopathy and Myosin Studies
Type
article
Field-Weighted Citation Impact
5.55

Funders

Controls
|||
ALL TIME
JAN
FEB
MAR
APR
MAY
JUN
JUL
AUG
SEP
OCT
article

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

Sherif F. Nagueh, Fatbardha Varfaj, Kathryn N. Ivey, John R. Giudicessi et al.
4 citations
Cardiovascular Research
Cardiomyopathy and Myosin Studies
5.55
article

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

Sherif F. Nagueh, Fatbardha Varfaj, Kathryn N. Ivey, John R. Giudicessi, Milind Y. Desai, LaTanya Tomlinson, Matthew J. Pollman, Laura M. Lombardi, Linda M. Yamamoto, Michael J. Previs, Gretchen Argast, Richard Pushkin, Brian Mangal, Danielle Kellner, W. G. Harrison, Whittemore G. Tingley, Laura Robertson, Natasha Sonicheva-Paterson
article en
4 citations

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.

Cardiovascular ResearchVol. 121(17)
University of Vermont (US), Houston Methodist (US), Cleveland Clinic (US), Rapt Therapeutics (United States) (US), Methodist Hospital (US), Mayo Clinic in Arizona (US)
Brigham and Women's Hospital, Cincinnati Children's Hospital Medical Center, Cleveland Clinic, Houston Methodist Hospital, Houston Methodist Research Institute
Openalex Percentile: Top 3%
Cardiomyopathy and Myosin Studies
5.55
DOIOpen Access PDFOpenAlex
AI Navigator

Ask Laika to Summarize, Analyze, and Connect papers live on the map.

Summarize Papers & Methodologies

Extract key findings, datasets, and comparative methods across publications.

Benchmark Rankings & Visual Analytics

Rank top research institutions, authors, funders, topics, and journals by Field-Weighted Citation Impact (FWCI) and paper volume with instant charts.

Connect Distant Disciplines

Bridge topological clusters on the map to find hidden collaborative intersections.