AAV-mediated gene therapy for Rare Bleeding Disorders
Rare inherited bleeding disorders (RBDs), include deficiencies of coagulation factors fibrinogen, factor (F)II, FV, combined FV and FVIII, FVII, FX, FXI, FXIII, and deficiency of vitamin K-dependent factors. Of all RBDs, the most in need of treatment are FVII- and FX-deficient patients. In our ongoing Health Holland, the RBD-Cure project we are working on the development of gene therapy for FVII and FX deficiency. Furthermore, we are aiming to develop platform technology which can also improve the expression of our gene therapy products as well being applied to other monogenetic diseases.
SanaGen was incorporated in 2019 and is headquartered at the New West Health & Innovation District in Amsterdam. SanaGen focuses on gene therapy approaches for rare genetic disorders that can provide a life-long cure for many diseases. SanaGen develops these new gene therapies in collaboration with the research group of Prof. dr. Jan Voorberg of Sanquin Research and Prof. dr. Joost Verhaagen of The Netherlands Institute of Neuroscience. The current portfolio includes the development of gene therapy for patients with factor VII and X deficiency. These rare bleeding disorders affect one in 300,000-500,000 people and current treatment for these disorders are suboptimal. Building on our extensive knowledge in the field of hemostasis as well as gene therapy development we are also focusing on gene therapy approaches for other bleeding disorders.
Davis Mosmuller | CEO at SanaGen
David Mosmuller is a medical doctor with a business mindset who has extensive experience in team management, clinical research, life sciences, medicine, regulatory affairs, and pharmacovigilance. He has a PhD in medicine from the VU University Amsterdam and worked in the past within the medical affairs departments of Sanquin Plasma Products and Novo Nordisk in the Netherlands. With SanaGen he aims to disrupt the market by developing affordable gene therapy products for patients with rare bleeding disorders and making these therapies globally available.
Bas Blits | Principle Scientist at SanaGen
Bas Blits is a scientist with 20+ years of experience in the field of gene therapy. After obtaining his PhD on gene therapeutic strategies for spinal cord repair he went for his Postdoctoral training to the Miami Project to Cure Paralysis before joining AMT (currently UniQure), where he spent 10 years of his career. At UniQure he was head of the neurobiology research department with several research programs in the pipeline up till the preclinical stage. He later worked at DegenRx, developing a gene therapeutic strategy against amyloid beta oligomers for Alzheimer’s Disease and is currently also working part-time at Crossbeta Biosciences for Parkinson’s disease. At SanaGen, Bas is responsible for the research and development of gene therapy for rare bleeding disorders.
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