Overcoming FSHD together

At Facio Therapies, we have a single focus - to find a cure for FSHD. More than 700,000 people worldwide are suffering from this progressive muscle wasting disease. Currently there are no treatment options available for people with FSHD. Arising from and working in alignment with the FSHD community, we are developing a therapy to tackle the cause of FSHD and to stop the progression.

Infographic overcoming FSHD together
No Dux4

The key event in FSHD is the undesired production of the DUX4 protein in muscle cells, causing them to waste away. We are developing a therapy to repress the production of this protein.

Plus SMCHD1

Our approach

The key event in FSHD is the unwanted production of a protein called DUX4. When produced in muscle tissue, DUX4 is highly toxic due to a cascade of events eventually resulting in the devastating effects of FSHD. In people without FSHD, the production of DUX4 is repressed by regulatory mechanisms in the muscle cell. So this is what a causal FSHD therapy should do: restore that repression as much as possible.

Our R&D

Discovering effective ways to repress and prevent the production of DUX4 is extremely challenging because of DUX4’s naturally elusive behavior and the many technical hurdles for its detection in muscle. In collaboration with our partners, we have established the first-ever validated screening platform based on the detection of the naturally occurring DUX4 protein in muscle cells from FSHD patients. By overcoming this challenge, we have created an essential platform for the discovery of innovative approaches to tackle the cause of FSHD.

In fact, we screened over 34,000 small molecule compounds, and obtained over 300 so-called ‘hits’ and identified novel approaches to repress the production of the muscle-toxic DUX4 protein. However, these are no medicines for FSHD yet. Thereto, we carefully evaluate how they work in FSHD to prioritize and optimize avenues with the most suitable safety and efficacy profile. In addition, we are preparing for the development of lead compounds for preclinical and clinical development. This way, we are working towards a much needed treatment for FSHD, without cutting corners.

Transparent Costs

Our business philosophy

With our single focus on FSHD, we devote our capital to doing only what is needed to translate recent scientific advancements into a causal therapy. That way, we are able to pursue a socially responsible business model that creates value for patients as well as shareholders. Consistent cost management will enable us to maximize therapy access by making it affordable and reimbursable. Also, we will reinvest part of our future profits into projects to expand quality-of-life options. Because we hold ourselves accountable to the FSHD community, we will be transparent on our plans, achievements, and future patient access strategies.

Alignment with the FSHD community

We are for and by people with FSHD. Our founders are three members of the FSHD community: business leaders Kees van der Graaf (Netherlands), Bill Moss AO (Australia), and Neil Camarta (Canada). We are funded by FSHD-affected families and friends from Australia, Europe, and North America, by the FSHD Global Research Foundation (Australia), the (Dutch) FSHD Stichting, the FSHD Canada Foundation, and Amis FSH (France), and by our drug discovery partner, Evotec (Germany). With the support from people with FSHD and their families and friends, we are determined to overcome FSHD together.

Our team

Our team

We are a growing multidisciplinary team with many years of pharmaceutical drug discovery & development experience. Combining this with the unstoppable drive from leading members of the FSHD community, gives us a brainy passion to break through to a therapy for FSHD.

Meet our team

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FSHD

Overcoming FSHD

Overcoming FSHD together