What we want to change:
Unaffordable gene therapies
There are already gene therapies on the market for a number of rare diseases. The one-time treatments we are developing—the so-called lentiviral gene therapies—are currently offered by companies at prices of as much as €2 to €3 million per patient.
Because developments are moving so quickly, many more gene therapies are expected to become available in the coming years. At these prices per patient, this will be unaffordable for society.
Health insurers and government authorities are therefore raising the alarm more and more often. This creates uncertainty for companies about whether future therapies will still be reimbursed.
This uncertainty is already causing several biotech and pharmaceutical companies to stop further development of gene therapies for certain rare diseases. As a result, an increasing number of promising potential treatments never reach patients.
Time for change
We will bring an affordable alternative to market
Where the traditional model aims to maximize profits, our model focuses on making treatments available to every patient. At transparent costs and socially acceptable margins—solely to ensure the continuity of our company and enable future developments. That means no profit distribution to shareholders.
Read how our model differs from the traditional model
We start with Pompe disease
For the treatment of Pompe disease and Hunter syndrome, medications are already being used: so-called enzyme replacement therapy. This therapy is extremely expensive—hundreds of thousands of euros per patient per year. We are developing our lentiviral gene therapy with the aim that, after a single treatment, this costly enzyme replacement therapy can be fully or almost entirely eliminated. This would result in enormous healthcare cost savings.
Why we start with Pompe disease
What research has been conducted?
The following websites explain the current status of our research:
On the path to a clinical trial for Pompe disease – Spierfonds
Closer to a treatment for Hunter syndrome – Brain Foundation
Only possible with societal support
We are a public company: a 100% subsidiary of Erasmus MC. We have a social mission that we adhere to. Like any academic hospital, Erasmus MC is largely funded by taxpayers and health insurance premium payers. We also receive funding from the Prinses Beatrix Spierfonds—money donated by supporters. In short, society pays, and we want to handle that money carefully and efficiently.
Will you help us?
We also receive funding from the Prinses Beatrix Spierfonds—money donated by supporters. You can help us by making a donation:
Would you like to contribute to our research in another way?
More to explore
How LentiCure’s model differs from the traditional model
Tuesday, January 20, 2026
The traditional model of drug development focuses on profit, which—especially in rare diseases—leads to high prices and limited availability. Our model takes a different approach and focuses on accessible treatments at transparent costs.
Why do we start with Pompe disease?
Thursday, January 22, 2026
Bringing the first innovative lentiviral gene therapy to market is the most expensive step. For a second therapy, much of the existing knowledge, data, and infrastructure can be reused, making the development process less costly—and this effect increases with each subsequent therapy. That is why it is essential that the first therapy we develop not only improves patient outcomes, but also reduces healthcare costs. Only then can we generate the financial resources needed to make further development possible.
Laatste nieuws
Podcast: Research is the key – Let’s Talk Business
Tuesday, January 20, 2026
The Prinses Beatrix Spierfonds funds, guides, and stimulates scientific research into muscle diseases, because it knows that scientific research is the only key to making all muscle diseases treatable in the future. Listen to the New Business Radio podcast Let’s Talk Business #634.
Donate to the Spierfonds and make a difference
Tuesday, January 20, 2026
Every day, people with a muscle disease need our help. With your donation, you help make groundbreaking research possible, improve treatments, and give hope to thousands of people in the Netherlands. Discover why your contribution can have such an impact.
We have never been more hopeful
Tuesday, January 20, 2026
Start-up for gene therapy for Pompe disease. With the collaboration between Erasmus MC and the start-up LentiCure, the Prinses Beatrix Spierfonds is taking a new step. The goal is to develop a gene therapy for Pompe disease.