SanaGen, at the forefront of universalising genetic treatments for ultra-rare diseases

SanaGen was incorporated in 2019 and is based in the New West Health & Innovation District in Amsterdam, the Netherlands. The company focuses on gene therapy approaches for rare genetic disorders and excels at exploring creative ways to reduce development costs, as well as developing innovative sales and contracting models to help bring genetic treatments to low-income patients and patients with ultra-rare diseases. Recently, GenScript Biotech‘s Europe’s marketing specialist, Nicholas Gouw, had the opportunity to speak with two of its representatives, the company’s co-founder and CEO, David Mosmuller, and Bas Blits, principal scientist at SanaGen.

Both share a humanitarian vocation that is in the company’s DNA and conditions its areas of interest and activity. “We focus on rare diseases because we truly believe that all patients should have access to treatment, not just patients with a disease that is more common. Our first target is rare bleeding disorders, and that has to do with our background,” said Dr. Mosmuller, who came into contact with these diseases while working at Sanquin plasma products, the plasma fractionation company of the Dutch Blood Bank, where he began to nurture the idea of creating a company specialised in genetic treatments for these types of blood diseases.

Currently, Sanquin and the Netherlands Institute for Neuroscience (NIN) are the company’s main collaborators in a project to develop adeno-associated viral vector (AAV)-mediated gene therapy for factor VII and factor X deficiency in blood, ultra-rare diseases that affect between 1 in 300,000 and 1 in 500,000 people. The project is led by Bas Blits, SanaGen’s principal scientist for the past year and a half, who is also a member of the NIN and an expert in the development of viral vector treatments.

“We have done a lot of work on this gene therapy platform, and I personally have been working for more than 20 years in the field of gene therapy with all kinds of vectors: adenoviral vectors and lentiviral vectors. But I think the most promising ones are adeno-associated viral vectors. At NIN we have a lot of experience in the production of recombinant AAV vectors (…) we produce AAV vectors in-house and that gives us the flexibility to do research in developing new vectors and try new things,” he explains.

Although currently focused on bleeding disorders, the young company has the ambition to expand to other rare disorders in the future, and thus they are already working together with their partners on the development of a gene therapy platform with novel capsids and new promoters. “For this gene therapy platform, we intend to target the liver as an organ and, of course, any liver-related disease, and we are investing a lot of time and effort in developing the ingredients for AAV-mediated gene therapy, which promises to be remarkably better compared to existing treatment options today,” remarks Mosmuller.

To further expand the capacity of its treatments, SanaGen needs additional funding to what it currently receives, limited to public grants plus a loan from the North Holland Innovation Fund. “At the moment it’s all public money and there is no private money, but now we are looking for private funding, because we need enough funding to go to the next stage (…) which means we are always looking for and talking to investors,” David explains, lamenting the limited funding available in the biotech field.

“Of course, this is because biotechnology is a high-risk investment. There is always a high probability that things will not succeed and the minimum investments needed are high (…) but that is not a reason to forget about these diseases. I think it’s great to be there for these patients and to get these treatments to the market, but we need ample funding to do that,” explains the SanaGen CEO, who calls for a paradigm shift in investment models to get more funding for a sector whose results and benefits are generally much longer term than in other areas.

“I really hope that more types of investors and investment opportunities will develop, because what I see right now is that only traditional biotech funds are interested in companies like SanaGen and the like. We -as a society- need to invest more in healthcare, and not only in biotech, but also in developing new drugs, new vaccines and in all other areas, because without good healthcare and good health, we are lost,” he says.

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