High rate of seroeligibility among MYBPC3-associated hypertrophic cardiomyopathy patients for TN-201, an adeno-associated virus serotype 9 MYBPC3 gene therapy
Pre-existing immunity to AAV9 was absent or low in most <i>MYBPC3</i>-associated HCM patients, with only a small proportion exceeding standard NAb thresholds for AAV gene therapy trials. These findings support the feasibility of a clinical trial of TN-201, an AAV9-based <i>MYBPC3</i> gene replacement therapy, in this population. Given the concordance between NAb and TAb assays, either may be suitable for screening.
Authors
- Sherif F. Nagueh (ORCID: https://orcid.org/0000-0002-4911-8056)
- E. Flores (ORCID: https://orcid.org/0000-0002-6100-8183)
- Omar Wever‐Pinzon (ORCID: https://orcid.org/0000-0002-2828-707X)
- Theodore P. Abraham (ORCID: https://orcid.org/0000-0001-8966-6906)
- Daniele Massera (ORCID: https://orcid.org/0000-0002-9499-6456)
- John R. Giudicessi (ORCID: https://orcid.org/0000-0001-5871-0265)
- William Harrison (ORCID: https://orcid.org/0000-0002-8214-1522)
- Milind Y. Desai (ORCID: https://orcid.org/0000-0003-0010-9797)
- Natalia Sonicheva‐Paterson (ORCID: https://orcid.org/0000-0001-7766-1251)
- Timothy C. Wong (ORCID: https://orcid.org/0000-0002-1045-2698)
- Emre E. Turer (ORCID: https://orcid.org/0000-0001-8782-3648)
- Ahmad Masri (ORCID: https://orcid.org/0000-0002-6390-6526)
- Gretchen Argast
- Florian Rader (ORCID: https://orcid.org/0000-0003-1637-5096)
- Pfumo Mushonga
- Heng Wang (ORCID: https://orcid.org/0000-0002-3563-2337)
- Neal K. Lakdawala
Institutions
- Cedars-Sinai Medical Center (US)
- Brigham and Women's Hospital (US)
- Houston Methodist (US)
- Cleveland Clinic (US)
- Oregon Health & Science University (US)
- University of California, San Francisco (US)
- NYU Langone Health (US)
- Center For Children With Special Needs (US)
- Nagase ChemteX (Japan) (JP)
- Mayo Clinic in Arizona (US)
- University of Pittsburgh Medical Center (US)
- Advanced Imaging Research (United States) (US)
- The University of Texas Southwestern Medical Center (US)
Publication Details
- Journal
- Frontiers in Medicine
- Published
- 2025-09-12
- DOI
- https://doi.org/10.3389/fmed.2025.1635586
- Primary Topic
- Virus-based gene therapy research
- Type
- article
- Field-Weighted Citation Impact
- 0.00