Facio screens 34,000 compounds
December 28, 2016
Facio Therapies announced today that it has screened approximately 34,000 compounds for their ability to repress the production of the muscle-toxic DUX4 protein in cultured muscle cells derived from FSHD patient biopsies. As planned, a variety of compounds with the desired effect (known as “hits”) have been identified.
In September 2016, Facio announced that, together with its drug discovery partner, German-based Evotec, it had established the first-ever screening platform that enables reliable quantification of naturally occurring DUX4 protein in cultured FSHD-affected muscle cells. Undue production of DUX4 is the cause of FSHD. 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. Facio’s single goal is to pursue a therapy that restores this repression as much as possible.
“This is the very first large scale FSHD screening campaign on record that quantifies the naturally occurring DUX4 in patient derived skeletal muscle cells”, noted David Dasberg, Facio’s Managing Director. He added: “Hits are the foundation for the development of our FSHD drug candidates, so we have to be very thorough. That’s why we are now subjecting all our hits to a battery of additional tests to weed out false positives. We look forward to completing this process in the months ahead.”
About Facio Therapies
Arising from the FSHD community, Facio is translating 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.