Novel curative treatment for rare bleeding disorders

The RBD‐CURE project provides a public private partnership that aims to develop innovative therapies for rare bleeding disorders. The unique expertise of Sanquin on bleeding disorders and expression of blood coagulation factors combined with the advanced gene therapy strategies developed at the Netherlands Institute for Neurosciences and SanaGen will be used to develop curative therapies for patients with bleeding disorders in both developing and non‐developing countries.

Over 500.000‐1.000.000 patients worldwide are suffering from rare bleeding disorders. Especially in the developing countries virtually no treatment is available for these patients. The majority of patients with rare bleeding disorders suffer from hemophilia A or B. A smaller proportion of patients lack blood coagulation factor VII, XI, X, prothrombin, fibrinogen or factor XIII. Current treatment for these patients is composed of blood plasma derived products that need to be infused repeatedly. Due to its high overall costs this treatment is available only for a limited number of patients. In the current project innovative gene therapy‐based approaches for treatment of rare bleeding disorders will be developed using engineered AAV variants that preferentially target the liver. The combined use of highly efficient liverhepatocyte‐ specific expression modules and capsid engineered AAV variants is expected to generate a versatile gene therapy platform that can be used to develop highly efficient curative therapies for rare bleeding disorders.