Developing new gene therapies

    SanaGen is a pioneering company that focuses not just on gene therapy, but also on exploring creative ways to reduce development costs, with an intent to introduce unique sales and contracting models for getting products to patients, and production built to serve both developed and developing countries.

    Why focus on Gene Therapy?

    If you ask patients what they want in a therapy, they usually respond with the same answer: They want a long lasting and durable cure, so that they can live their lives free from their disease. For societies, the answer is the same, a cure which allows freedom from a disease but also doesn’t have high, reoccurring therapy costs like chronic therapy treatment. Gene therapy is an ideal solution for both; the best for the patient, and the best for society.

    Monogenetic Disorders are Ideal Gene Therapy Candidates

    Many monogenetic disorders cause a lack of a specific functional protein in a person. This leads to a lifelong disease, where treatment, if it exists today, often requires the replacement of the protein intravenously or with some other replacement biologic therapy. This is expensive and inconvenient for the patient. Gene therapy offers the promise to end this compromise.

    Gene therapy

    SanaGen was incorporated in 2019 and is headquartered at the New West Health & Innovation District in Amsterdam, The Netherlands. SanaGen focuses on gene therapy approaches for rare genetic disorders that can provide a life-long cure for many diseases. SanaGen is a pioneering company that focuses on transparency of costs and exploring creative ways to reduce development costs, as well as on innovative sales and contracting models for getting products to patients, and production built to serve both developed and developing countries. 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.

    Our 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. Over 3 decades of gene therapy research and experimentation have led to a rapidly innovating industry which is on the brink of treating many different genetic disorders. The principal founders of SanaGen are experts in both basic and clinical studies on bleeding disorders and also include experts with detailed knowledge on global markets for these disorders.

    SanaGen is founded and headed by David Mosmuller, MD PhD. He is a medical doctor with a business mindset with extensive experience in clinical research, life sciences, medicine, regulatory affairs, and pharmacovigilance.

    About us

    SanaGen was incorporated in 2019 and is headquartered at the New West Health & Innovation District in Amsterdam, The Netherlands. SanaGen focuses on gene therapy approaches for rare genetic disorders that can provide a life-long cure for many diseases. SanaGen is a pioneering company that focuses on transparency of costs and exploring creative ways to reduce development costs, as well as on innovative sales and contracting models for getting products to patients, and production built to serve both developed and developing countries. 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.

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    Gene therapy

    The overall goal of gene therapy for rare bleeding disorders, including factor VII, VIII, IX and X deficiencies, is to provide patients with functional factor genes capable of producing the respective proteins that are missing in the patient. In principle, this may be achieved by gene transfer, whereby genetic material is delivered using viral vectors or non-viral means, or by gene repair/editing, in which the causative mutations are identified and subsequently corrected. For over two decades, studies on gene therapy for hemophilia A and B have been performed and they are currently in phase III of clinical development, with the first approval expected by the end of 2022. By capitalizing on this knowledge, SanaGen is able to efficienctly and cost effectively develop new rare bleeding disorder therapies for unexplored markets.

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    Meet the team

    David Mosmuller

    MD, PhD, Co-Founder and CEO

    Matthew Hotchko

    PhD, MBA, Co-Founder and Board Member

    Bas Blits

    PhD, Principal Scientist

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    SanaGen: one step closer to a cure for genetic disorders