Rare

Worldwide, 500.000 to 1.000.000 people suffer from rare clotting diseases

Gene therapy for rare coagulation diseases

Worldwide, 500,000 to 1,000,000 people suffer from rare clotting diseases. Sanquin is working with the Netherlands Institute for Neuro Science and the Amsterdam-based start-up SanaGen on the development of gene therapy for the effective treatment of these diseases.

The Amsterdam-based start-up SanaGen, with former Prothya Biosolutions colleague David Mosmuller as director, is developing a gene therapy platform for use in the treatment of various coagulation diseases. People with such a disease have abnormalities in their DNA, as a result of which they do not produce sufficient, or any, coagulation proteins. Using gene therapy, you place synthetically manufactured genes in the liver cells, causing them to produce the missing coagulation proteins.

Life Changer

In this way, a successful treatment has already been developed for hemophilia B, for example. With a single injection patients can be free of this tendency to bleed for at least ten years, and they no longer require an infusion with coagulation factor several times a week. This treatment is therefore a real life changer.

Affordable and Accessible

It remains a problem that gene therapy is not being developed for very rare diseases or that it is only being made available at very high prices. Furthermore, 75% of patients worldwide with bleeding disorders (mainly hemophilia A and B) have no, or only limited, access to any effective treatment. The Sanquin-led consortium aims to develop an affordable gene therapy platform for several rare bleeding disorders, so that as many patients as possible can be treated worldwide.

More funding

Sanquin is in partnership with the Netherlands Institute for Neuro Science and SanaGen in this development project. We provide our expertise in the field of blood coagulation and are working on smart delivery systems for this new therapy.
The Netherlands Institute for Neuro Science and SanaGen in turn have a great deal of knowledge of gene therapy. The Innovatiefonds Noord-Holland (North Holland Innovation Fund) recently granted a loan of €300,000 to SanaGen for the further development of the gene therapy platform.

Continuous improvement

Professor Jan Voorberg, head of Sanquin Research’s Cellular Hemostasis Department, is actively involved in the development with his research group. Jan: “This cooperation fits in nicely with Sanquin’s objective of continuously improving patient care. In principle, gene therapy can be used to correct all genetic abnormalities. We expect to be able to start the first clinical trials in a few years’ time.”