LentiCure: Innovation in Gene Therapy for Rare Muscular Diseases. Erasmus MC spin-off company LentiCure develops an advanced and life-saving gene therapy for Pompe disease and bring it to the market at a socially responsible price
LentiCure is an ambitious start-up focused on life-saving gene therapy for rare muscular diseases such as Pompe disease and Hunter syndrome. As a spin-off of Erasmus MC, the leading knowledge center for Pompe disease, they collaborate closely with the Princess Beatrix Muscle Foundation.
Pompe Disease: A Rare Genetic Muscle Disorder
In the Netherlands, approximately 200,000 people suffer from Pompe disease, a rare hereditary muscle disorder in which the body produces insufficient amounts of alpha-glucosidase. This protein is essential for breaking down sugars within cells, and its deficiency leads to the progressive breakdown of muscle tissue. The most severe forms of the disease can also affect the heart and brain. Without treatment, babies with Pompe disease often die within a year.
Fortunately, a therapeutic option has been available since 2006, but this treatment is not equally effective for all patients and, critically, it does not reach the brain.
A Breakthrough in Gene Therapy
The team led by Pim Pijnappel, professor of Cell and Gene Therapy at Erasmus MC, has developed a gene therapy that can restore function in the heart, muscles, and brain with a single treatment. Here's how it works: first, stem cells are extracted from the bone marrow of the patient with Pompe disease. In those stem cells, the hereditary error is in the DNA that normally produces the alpha-glucosidaseprotein. Then a correct piece of DNA is inserted into the stem cells, so that they can produce the protein again. This is done by using a virus that does not make a patient sick. That virus works its way in with the missing piece of DNA, so that it can be built into the DNA which results in healthy cells. Finally, the corrected stem cells are returned to the patient's bone marrow.
World-Class Research in the Netherlands
Research into gene therapy for Pompe disease in the Netherlands is at ta leading level of global efforts. While other research groups have developed gene therapies for this condition, these therapies have limitations: they cannot reach the brain, cannot be used in children under four years old, and are expected to lose effectiveness over time. The new therapy developed at Erasmus MC aims to address these challenges with a one-time treatment that targets all muscles and organs, including the brain.
Accessible Healthcare: the Need for Affordable Therapies
While research is crucial for preventing and stop muscle diseases, an innovative approach is needed to make these therapies affordable for patients. This is a key focus of the collaboration between Erasmus MC, LentiCure, and the Princess Beatrix Muscle Fund. Together, they have united with a single goal: to bring an advanced, life-saving gene therapy for Pompe disease to market at a socially responsible price.
The idea of affordable medicines is not new, but the approach taken to achieve this goal is groundbreaking. At the heart of this effort is LentiCure’s disruptive business model, which seeks to reshape the landscape of healthcare accessibility.
LentiCure is a 100% daughter startup of Erasmus MC, funded entirely through private investments, with a vision to keep its capital in public hands. Its unique approach to managing private donations in a transparent, responsible, and efficient manner allows the company to keep development costs low, thereby making the therapy more affordable for patients.
Princess Beatrix Muscle Foundation
Princess Beatrix Foundation the biggest partner making non –precedential “investment” into a start-up company (LentiCure) plays a crucial role in connecting directly with the patient organizations securing an active participation of those groups in the process. This is indispensable in steering the developments of the therapy and serves to search and include other partners/partner organizations that contribute to the process.
Erasmus MC is carrying out the complex research efforts, including clinical trials, and serves as the sponsor for these trials.Erasmus MC collaborates with various regulatory authorities to secure approval for the therapy and works with external (medical) experts to bring the gene therapy closer to patients worldwide. As a renowned university medical center, Erasmus MC has built a strong reputation for treating rare diseases, both in the Netherlands and abroad.
Socially responsible licensing
The mission of the Technology Transfer Office (TTO) at Erasmus MC in this process was to facilitate this unique collaboration by aligning the interests and values of all partners. TTO's role included establishing terms that would make the therapy accessible to patients through socially responsible licensing, in line with the principles set forth by the NFU (Dutch Federation of Universities) and outlined in the license agreement between LentiCure and Erasmus MC. These principles include responsible pricing, ensuring a fair and balanced collaboration between stakeholders, and ensuring that the technology licensed to LentiCure is further developed and reaches the target patient group.
What are the effects of using the blue print business model on a short and longer term?
LentiCure’s strategy and licensed technology enable the company to categorize patients into smaller clusters based on the specific type of rare disease, allowing for the development of tailored therapies for each target group. In the short term, LentiCure’s focus is on Pompe disease—finalizing its development and delivering the therapy to patients at a fair price. This approach is designed to generate a financial buffer that can support the development of therapies for other rare diseases in the pipeline. At the same time, LentiCure is pursuing a dual innovation strategy: developing an effective, progressive gene therapy that is brought to market through a non-commercial route at a transparent price, while also laying the groundwork for the development of additional gene therapies for other rare diseases. In this way, LentiCure aims to serve as a springboard for future therapies for a wide range of rare diseases.
Director of this new venture Dirk van Asseldonk described the intent behind this different way of approaching a therapeutic academic spinoff: “We will not only be able to train foreign hospitals in gene therapy for Pompe disease, but also share a blueprint of how to bring such therapies to patients with a social company. If every university hospital offered gene therapy for a few rare diseases, we would go a long way together towards tackling these life-altering disorders.
