Facio enters into agreement with scientific leaders to advance its drug discovery program 

Leading academic institutes active in the field of FSHD have agreed to transfer materials to Facio for its drug discovery program aimed at stopping the progression of FSHD. 

Facio’s drug discovery program is designed to identify compounds showing activity as a potential treatment to stop the progression of FSHD. The Dutch based Leiden University Medical Center, department of Human Genetics led by Prof Dr Silvère van der Maarel (“LUMC”), Dr Stephen Tapscott, a full member of Fred Hutchinson Cancer Research Center’s Human Biology Division (“Fred Hutch”) and Prof Dr Rabi Tawil from the Neuromuscular Disease Center at the University of Rochester (“URMC”) will support this program by providing human FSHD-affected muscle cell lines and know-how. 

Together with Evotec, Facio recently started its drug discovery program to identify small molecules that increase the activity of SMCHD1 and restore the repression of DUX4. The results from this program are expected in the first half of 2016. 

Professor van der Maarel: “Our providing material to Facio supports the translation of the combined research on FSHD, performed by Dr Tapscott, Dr Tawil and myself, towards an FSHD therapy. I am really pleased working with the Facio team and look forward to the breakthrough towards a causal therapy for people with FSHD.” 

David Dasberg, managing director of Facio, commented: “We are very pleased to work together with these leading scientist in the field of FSHD, who have revealed the major role of SMCHD1 in the progression of this epigenetic disease. Together with our partner Evotec and with the support from Drs van der Maarel, Tapscott and Tawil we believe we can overcome FSHD together.” 

For more information on the Leiden University Medical Center and the department of Human Genetics, please visit LUMC 
For more information
 on Fred Hutch and the Human Biology Division, please visit Fred Hutch 
For more information on the University of Rochester and the MDA Neuromuscular Disease Center, please visit URMC