Rare Bleeding Disorders, Gene Therapies, and Treatment Accessibility – SanaGen

SanaGen focuses on gene therapy approaches for rare genetic disorders that can provide a life-long cure for many diseases.

Life Science interviews the CEO and Principal scientist of SanaGen, a company pioneering pioneering the development of gene therapies and innovative pricing models to make these treatments more affordable in less developed countries

“A higher price for genetic treatments in rich countries to fund them in poor countries would be a very nice model”.

David Mosmuller, MD, Ph.D. – Co-Founder and CEO, SanaGen
David Mosmuller, MD, PhD. & Bas Blits, PhD. (SanaGen); Featured speakers at Biotech Fast Forward 2021

Founded in 2019 and based in Amsterdam, SanaGen is a company focused on developing gene therapy models that can provide a lifelong cure for many rare genetic diseases and disorders. If the company had a motto to sum up its business philosophy, it could well be the maxim ‘gene therapy for all!” It may sound utopian, but this Dutch start-up stands out by exploring creative ways to reduce the costs of developing and marketing expensive genetic treatments, developing innovative sales and recruitment models to get products to patients, and targeting its production to both developed and developing countries. Recently GenScript Biotech‘s European Marketing Specialist, Nicholas Gouw, had the opportunity to speak with two of their representatives, the company’s co-founder and CEO David Mosmuller and Bas Blits, SanaGen’s Principal scientist.

As Mosmuller and Blits explained, the company’s current main area of interest is rare bleeding disorders, specifically those caused by deficiency of factors VII and X in the blood, both being rare diseases that cause blood clotting problems in between one in 300,000 and one in 500,000 people. To try to cure them, the company is developing an adeno-associated viral (AAV)-mediated gene therapy, led by Blits, a scientist with more than 20 years of experience in the field of gene therapy, in collaboration with the Netherlands Institute for Neurosciences (NIN) and Sanquin, the plasma fractionation company of the Dutch Blood Bank.

It was precisely while working at this company that the idea of founding SanaGen began to take shape in David’s mind. In the course of his work, with constant contact with patients and organisations, Dr. Mosmuller realised that despite the availability of budgets for diseases such as haemophilia A and B, the very high cost of treatments limited them in practice to high and upper-middle income countries. Moreover, economic issues were not the only obstacle to applying these treatments in less developed countries, where all sorts of “practical problems” arose. One example is the infusion of proteins into the blood, a treatment “that parents have to be taught to give to their young children until they can do it themselves”, or the lack of refrigerators, essential for storing some of these products.

“That’s when I thought: Ok, we have to come up with a new approach to gene therapy. Not only to cure these patients, but also to alleviate the practical burdens associated with current gene therapy options, because these practical burdens are the reason why 75% of patients with bleeding disorders worldwide have not, or have minimal access to the treatments needed for their disorder. We still face many challenges in terms of gene therapy pricing, but this can be offset to some extent by easing practical burdens and bringing treatment to larger scales. If you scale up globally, there are always more patients to consider than just in high-income countries, so there is always a need to consider innovative pricing models and see what solutions to the above problems can be discovered,” says David.


The problem of pricing

The philosophy of the company he started has both objectives as its main, core pillars. Faced with the pricing problems that are common in the pharmaceutical market, which is currently determined by the first “one or two high-income countries that can pay the costs”, Dr. Mosmuller and SanaGen have long been studying a wide range of proposals that could be applied to lower the costs of gene therapy in less developed countries.

One of them relates to the duration of treatment and payment by instalments. In most cases, gene therapies do not involve continuous treatments where products have to be replenished from time to time, but are one-off therapies whose effects should last at least ten years and ideally last a lifetime. “If this is the case, then I would say it is a very honest approach to look at the effect of a treatment, and to charge costs only as long as the treatment is effective. Of course, if the effect is lifelong, then you would have to agree to cease payments at some agreed point in time. So I think we should aim for a model where you pay only for as long as the therapy works, like a monthly or annual fee, rather than a large amount of money up front, and I know that other gene therapy companies are also looking at similar models,” says the SanaGen CEO.

For developing countries, he advocates looking at burden-sharing models to ensure the right of the most disadvantaged people to receive treatment. “For example, a higher price could be paid for treatment in a higher income country, and with that higher price, a part of the treatment could be paid for others in lower income countries. This could be a very nice pricing model, but I know it’s going to be very difficult to convince others to implement,” says Mosmuller, whose company works with Fair Medicine, an organisation that has been promoting projects to develop medicines collaboratively and at more affordable prices for years.

Solving logistical problems: designated treatment centres

In addition to the purely financial ones, there are also challenges related to cultural, legal and logistical factors. In this respect, one of Mosmuller’s proposals is to work with designated treatment centres. “As it is not necessary to infuse the therapy regularly, patients can be brought to the product rather than the product to the patients. In this way, designated treatment centres can be established to streamline logistics, materials storage and treatment delivery, making these therapies more acceptable and more financially viable,” he says. Another interesting aspect is the looming horizon with the emergence of new technologies such as CRISPR/Cas and their regulatory implications, he notes.

But while it may be possible to reduce their cost and make it fairer, the SanaGen CEO makes it clear that genetic treatments will never cost “a few thousand euros” at least in the near future, and believes it is important for patients to understand why they are so expensive. “In the pharmaceutical sector there is always the question: is this a fair return on investment? Often it takes big risks and huge investments for a certain project to come close to its goals… and there is always the risk that things don’t work out, for example if the drug is not approved to enter the market in the end, so you have to take this financial risk into account as well,” explains Mosmuller. For him, the lack of understanding of this reality is “one of the main problems that the pharmaceutical industry is facing today” as most of the time people do not understand why some products are expensive and therefore conclude that “it’s just for some companies to get rich”, he says.

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