Consortium

The Development of gene therapy for rare bleeding disorders is a collaboration project between the research group of Prof. dr. Jan Voorberg of Sanquin Research, Prof. dr. Joost Verhaagen of The Netherlands Institute of Neuroscience and SanaGen.

Prof. dr. Jan Voorberg, head of the Molecular and Cellular Hemostasis research group at Sanquin, is an internationally recognized expert in the field of bleeding disorders who has extensively studied rare bleeding disorders such as haemophilia and other blood clotting factor deficiencies.

Prof. dr. Joost Verhaagen and his research group perform basic and translational research with the aim to advance the field of restorative neuroscience and neurology. They focus on the cellular and molecular mechanisms that underly degeneration, regeneration and plasticity of the central and peripheral nervous system. Adeno-associated viral vectors (AAV) and lentiviral vectors (LV) are used at this lab to target the nervous system.

From left to right: Prof. Dr. Joost Verhaagen (NIN), Dr. David Mosmuller (SanaGen), Prof. Dr. Jan Voorberg (Sanquin)

Fair Medicine

SanaGen works together with Fair Medicine on new business models to bring gene therapy for a fair price to as many patients as possible, both within Europe and around the world. Our first focus lies with the ultra-rare diseases where the traditional pharmaceutical model fails. The development of gene therapy for rare bleeding disorders includes the development of gene therapy for factor X and factor VII deficiency, diseases where currently there is no company or organization worldwide that is publicly working on a potential cure.

Fair Medicine is the first cooperative medicine collective that aims for developing economically sustainable therapeutics for patients worldwide. As the number of expensive pharmaceuticals increases, the health care of vulnerable and ill people comes under pressure. At the moment, there are virtually no models according to which safe, effective and affordable pharmaceuticals can be produced with a transparent price structure. Fair Medicine is unique in this aspect, as it is the only foundation in the Netherlands that develops new models and reports on its activities in a transparent manner. Society as a whole needs new strategies that result in affordable and accessible medication to a greater extent than existing models. Fair Medicine Foundation works to achieve this goal by facilitating the development of safe, effective and affordable medication. Fair Medicine primarily chooses to work in areas where market forces are not successful. 

Funding

The Development of gene therapy for Rare Bleeding Disorders, also known as the RBD-Cure project, is a collaboration project between the research group of Prof. dr. Jan Voorberg of Sanquin Research, Prof. dr. Joost Verhaagen of The Netherlands Institute of Neuroscience and SanaGen. This collaboration project is co-funded by the PPP Allowance made available by Health~Holland, Top Sector Life Sciences & Health, to stimulate public-private partnerships. Top Sector Life Sciences & Health (LSH) supports innovative research and development realized by public-private partnerships (PPPs) of the Dutch LSH sector. The Top Sector brings together stakeholders in a shared, consolidated innovation infrastructure. By means of PPP Allowance, the Top Sector provides a financial instrument to help consortia consisting of research organisations, knowledge institutes, companies, and health foundations to realise their innovative ideas. For more information please visit: https://www.health-holland.com/calls/tki-match

Prior to the RBD-Cure project the collaboration between Sanquin, The Netherlands Institute of Neuroscience and SanaGen started with the X-Cure project, developing gene therapy for factor X deficiency. This project was also co-funded by the PPP Allowance made available by Health~Holland, Top Sector Life Sciences & Health. The RBD-Cure project is a continuation of the X-Cure project with the objective to develop an affordable gene therapy platform for RBDs which can also improve the expression of our FX gene therapy product as well being applied to other rare bleeding disorders.

Furthermore, SanaGen has received a convertible loan worth €300,000 from the Noord-Holland Innovation Fund for the development of gene therapy for rare bleeding disorders. The Noord-Holland Innovation Fund is an initiative of the Dutch province of Noord-Holland, University of Amsterdam, Amsterdam University of Applied Sciences, Amsterdam UMC, and Sanquin, with support from the European Union through the European Regional Development Fund. The Innovation Fund supports entrepreneurs in the Dutch province of Noord-Holland by financing innovation in the Proof-of-Concept phase by providing convertible loans.