Gene therapy for rare bleeding disorders

Over 500.000-1.000.000 patients worldwide are suffering from rare bleeding disorders. Sanquin collaborates with the Netherlands Institute for Neurosciences and the Amsterdam-based start-up SanaGen to develop advanced gene therapy strategies for effective treatment of patients. 

The Amsterdam based start-up SanaGen, which was founded by former Sanquin-colleague David Mosmuller, is developing a gene therapy platform for treatment of rare bleeding disorders. Rare bleeding disorders are caused by genetic defects in the genes that encode blood coagulation factors. Liver-directed gene therapy can be used to correct the bleeding tendency in these patients.  

Lifechanger

Using this approach a successful treatment for hemophilia B has recently been developed. Long-term follow-up indicates that single infusion prevents bleeding complications in these patients for at least 10 years. This positions this novel treatment as a potential life-long cure. 

Affordable and accessible

Gene therapy for rare genetic disorders are not widely available and usually expensive. Worldwide 75% of the patients with bleeding disorder does not have access to adequate treatment. The Sanquin-led consortium aims to develop an affordable gene therapy platform that provides access to treatment for patients in both high- and low-income countries. 

Loan for SanaGen

Sanquin has partnered with the Netherlands Institute of Neurosciences and SanaGen. Sanquin provides expertise on blood coagulation and is involved in design of innovative delivery-systems for this novel therapy. The Netherlands Institute for Neurosciences and SanaGen bring in their know-how on gene therapy. Innovatiefonds Noordholland has granted a loan of 300.000 euro to SanaGen to support the development of this novel gene therapy platform. 

Continuously improving

Professor Jan Voorberg, head of the Department of Cellular Hemostasis of Sanquin Research, and his group are actively involved in this project. Jan: “The current collaboration fits well in Sanquins strategy to continuously strive for improving treatment for patients with blood-related disorders. Gene therapy can potentially provide a cure for monogenetic disorders. We hope that we will be able to initiate clinical studies using our novel platform in a few years.”