When blood flow to the heart is reduced — a condition called myocardial ischemia — it can cause chest pain, dizziness, and fatigue, and may progress to a heart attack. Current treatments only relieve symptoms without stopping the decline toward heart failure.
The adult heart has a limited ability to regenerate, replacing only about 0.5–1% of its muscle cells each year, insufficient to repair the damage caused by a heart attack. Fueled by recent discoveries demonstrating that existing heart muscle cells can be activated to proliferate, we are developing gene therapies designed to elicit true cardiac regeneration. These programs harness the power of RNA therapeutics, which can precisely modulate gene expression within heart muscle cells.
To protect RNA therapeutics from degradation in the human body and to ensure their precise uptake into heart muscle cells, advanced delivery tools are essential. We are therefore focused on developing heart-specific gene therapies with controllable performance to restore heart function safely and effectively.
AAV Vector Technology
Gene therapy with adeno-associated virus (AAV) vectors unlocks the potential to repair the heart from within. The challenge and opportunity lies in striking the right balance: activating heart muscle regeneration while maintaining precise control of gene expression. To achieve this, we are pioneering novel RNA- and protein-based technologies that enable on-demand, finely tuned therapeutic activity.
