Safety and Efficacy of Induced Pluripotent Stem Cell-derived Engineered Human Myocardium as Biological Ventricular Assist Tissue in Terminal Heart Failure

Summary

The BioVAT-HF trial will test the hypothesis that cardiomyocyte implantation via engineered heart muscle (EHM), the proposed investigational medicinal product (IMP; designated "Biological Ventricular Assist Tissue" or BioVAT), results in sustainable remuscularization and biological enhancement of myocardial performance in the failing heart. EHM are constructed from defined mixtures of induced pluripotent stem cell (iPSC)-derived cardiomyocytes and stromal cells in a bovine collagen type I hydrogel. Comprehensive preclinical testing confirmed the rationale for the clinical translation of the myocardial remuscularization strategy by EHM implantation. The patient target population for EHM therapy is patients suffering from advanced heart failure with reduced ejection fraction (HFrEF; EF: ≤35%) and no realistic option for heart transplantation.

Timeline

Start

2020-02-03

Primary completion

2027-12

Completion

2027-12

Publications

• Zimmermann WH, Ensminger S, Kutschka I, Paitazoglou C, Seidler T, Brandenburg S, Anker SD, Bader N, Bergau L, Bremmer F, Diogo PG, Eitel I, Fujita B, Gerecke B, Hasenfuss G, Hellenkamp K, Hermann-Lingen C, Jebran AF, Jurczyk D, Knaus R, Legler T, Lotz J, Placzek M, Puhler T, Riggert J, Sadlonova M, Saraei R, Strobel P, Tiburcy M, Ullrich C, Voigt JU, Walker F, Wollnik B, Yigit G, Friede T; BioVAT-HF Investigators. Stem-Cell-Derived Biologic Ventricular Assist Tissue in Heart Failure. N Engl J Med. 2026 May 28;394(20):1991-2001. doi: 10.1056/NEJMoa2513525.
• Montague EC, Ozcan B, Sefton E, Wulkan F, Alibhai FJ, Laflamme MA. Human pluripotent stem cell-based cardiac repair: Lessons learned and challenges ahead. Adv Drug Deliv Rev. 2025 Jul;222:115594. doi: 10.1016/j.addr.2025.115594. Epub 2025 May 5.

Drugs

Indications