Facio develops breakthrough tool for FSHD drug discovery
Facio Therapies has established the first-ever validated platform that enables screening large numbers of compounds for their ability to repress the production of the DUX4 protein in cultured muscle cells derived from FSHD patient biopsies. With this tool, Facio expects to identify series of compounds with the desired effect before the end of 2016.
Facio’s single focus is on developing a causal FSHD therapy. The key event in FSHD is the unwanted production of the DUX4 protein. 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. Therefore, Facio pursues a therapy that restores this repression as much as possible.
“To the best of our knowledge, this is a first”, said David Dasberg, Managing Director of Facio. “The reliable quantification of naturally occurring DUX4 in FSHD-affected skeletal muscle cells is extraordinarily difficult because it is an elusive protein with very low and variable expression levels. This challenge has, in fact, made large-scale screens on DUX4 impossible – until now. We are proud to have overcome this challenge in little more than a year.”
“By screening on DUX4 in a system that captures the natural complexity of FSHD, we maximize the potential to discover compounds that will be clinically meaningful,” added Kees van der Graaf, Chairman of Facio. “We therefore believe our platform represents a major leap for the FSHD community, from which we originate and to which we hold ourselves accountable.”
Facio expects to identify series of compounds with the desired effect (known as “hits”) over the next few months. These hits will undergo further extensive testing in order to generate so-called lead compounds suitable for development into a human therapeutic. Facio conducts its drug discovery program in close collaboration with Evotec International (Germany) and with the support of its academic partners.
About Facio Therapies
Arising from the FSHD community, Facio aims to translate breakthrough results of 25 years of basic research into a therapy that stops the progressive muscle wasting caused by FSHD. That is Facio’s product mission. Facio also aims to make its therapy widely available by using a transparent model for fair and reimbursable pricing. That way, Facio aims to generate sustainable profits to fund additional quality-of-life projects for people with FSHD and to provide liquidity to its shareholders. That is Facio’s socio-economic mission.