Next generation regulated AAV vectors for cardiac gene therapy

This project brings together leading scientists and a biotech company in a new public-private partnership to develop a controllable gene therapy for heart disease. By combining expertise and infrastructures, the project aims to deliver a next-generation regenerative therapy that is both safe and effective, with the potential to repair damaged heart tissue.

Heart disease remains the number one cause of death worldwide and is responsible for nearly 40,000 deaths each year in the Netherlands alone. After a heart attack, many patients develop irreversible heart damage that can lead to heart failure, a chronic condition with high healthcare costs and major impact on quality of life. Current treatments can only slow disease progression, they cannot repair the heart. Innovative therapies that directly repair heart tissue are urgently needed to improve survival, reduce hospital admissions, and lower long-term healthcare costs.

Our project focuses on developing a cardiac gene therapy that can be precisely switched on or off when needed. Unlike existing approaches, this therapy will allow doctors to control the duration of treatment, reducing serious side effects. The project combines cutting-edge genetic engineering, drug-responsive control systems, and advanced laboratory heart models to create a therapy tailored to the complexity of heart disease.

By the end of the project, we aim to deliver proof that controllable gene therapy is both safe and feasible for treating heart disease. This will include newly developed gene therapy switches, validated treatments in human-relevant experimental models, and a roadmap for clinical translation. Together, these outcomes will pave the way towards curative therapies that not only save lives but also reduce the economic burden of heart disease on society.