Top Sector Life Sciences & Health (LSH) entails a broad scope of disciplines, from pharmaceuticals to medical technology and from healthcare infrastructure to vaccination. To realise its mission – vital citizens in a healthy economy - the Top Sector builds on the strengths of the Dutch LSH sector to address the biggest societal challenges in prevention, cure and care. By funding multidisciplinary public-private partnerships (PPPs) the Top Sector aims to facilitate innovation. Here we give an overview of  a number of funded R&D projects by Top Sector LSH. The page is updated continuously.

Biological pacemaker made from lentiviral gene therapy

Lentiviral biological pacemakers that express HCN2/SkM1

In LentiPace, PacingCure B.V. works with the Amsterdam University Medical Centers, and Medtronic Bakken Reserac Center, on developing gene therapy-based biological pacemakers for patients with bradycardia (slow heart rate disorders).

Current state-of-the-art treatment is implantable electronic pacemakers which, while having gone through more than 50 years of evolution, still come with many problems that impact negatively on the lives of millions of patients who need a pacemaker. These problems are challenging to solve because they stem from the fundamental design of electronic pacemakers. Our technology leaps beyond state-of-the-art, finally allowing us to solve these problems by replacing electronic pacemakers with a hardware-free, biological treatment. The novel therapy provides improvements in quality of life (generating robust sensitivity to autonomic modulation, optimal cardiac output, and is devoid of hardware-related complications), at a reduced cost to society.

The biological pacemakers help to restore electrophysiological properties required for the maintenance of adequate and efficient cardiac contractions. It utilises gene therapy vectors to deliver therapeutic ion channel proteins to restore the function of the heart’s natural pacemaker and conduction system. This gene therapy product will be administered via a minimally invasive, transvenous procedure, employing clinically approved injection catheters, directly intramyocardially. By using lentiviral-based vectors, pacemaker function can exhibit a permanent (i.e., life-long) effect, carrying the potential to cure the diseased pacemaker and conduction system. The result is a more effective, more durable, and safer treatment whose monitoring requires a less complex and costly infrastructure and can be easily achieved from the patient’s home.

This joint effort builds further on earlier published animal models that demonstrated clear proof-of-concept and forms the missing link in the transition towards implementation of a long-term expression system (i.e. the lentiviral vector system). Such transition is crucial in the development of long-term clinical applications.