Facio presents at the 6th annual Orphan Drugs and Rare Diseases Europe ConferenceLeiden, the Netherlands - May 5, 2017
Facio Therapies will present its mission to improve the lives of people with FSHD, and showcase the latest results from its FSHD drug discovery program at the 6th annual Orphan Drugs and Rare Diseases Europe Conference in Berlin, Germany, on May 15th, 2017.
The 6th annual Orphan Drugs and Rare Diseases Europe Conference, is organized by SMI and connects the different stakeholders in the rare disease industry including patient advocates, regulators, pharma, investors and academia. Facio’s Managing Director, David Dasberg, will discuss Facio’s origin, the translation of science into early stage drug discovery, as well as community alignment. Facio’s Case Study: “Breaking Through to an FSHD Therapy” will be held at the Crowne Plaza Berlin – Potsdamer Platz, Berlin, Germany on May 15th, 2017 at 16:30 (CEST).
FSHD (facioscapulohumeral dystrophy) is a skeletal muscle wasting disease that devastates the lives of over 700,000 people worldwide and those close to them. The loss of muscle strength has a huge impact on daily life. Living with FSHD means living with pain, fatigue, and social isolation. Above all, the future becomes uncertain because the course of the disease is unpredictable. About 20% of people with FSHD end up in a wheelchair. Currently, no therapy for FSHD is available other than forms of temporary symptomatic relief.
About Facio Therapies BV
Facio Therapies, established in 2014, is a Netherlands-based company with a single focus: to overcome FSHD by developing a causal therapy that restores the natural repression of the muscle-toxic protein, DUX4. When unduly produced in skeletal muscle, DUX4 sets in motion a cascade of cellular events that eventually result in the muscle wasting seen in FSHD. Facio is the only one in the FSHD field with a fully automated, high-throughput screening platform based on quantifying the DUX4 protein in unadulterated (“primary”) FSHD-affected muscle cells. Facio delivered the first-ever therapeutically relevant proof of principle in FSHD by showing that one of its lead candidates, an orally active small molecule, represses DUX4 levels produced by human FSHD-affected muscle cells in a unique animal model (“in vivo”).
Rooted in, and dedicated to working for the FSHD community, Facio’s business approach is to have a positive impact on lives rather than to maximize financial gain. Since inception, Facio has raised over €16M in equity funding from FSHD-affected families, their friends, FSHD foundations, and Facio’s drug discovery partner, Evotec. Facio’s Board consists of business leaders from the FSHD community – Kees van der Graaf (Chairman; Netherlands), Neil Camarta (Canada), Dave Mackay (USA), Bill Moss (Australia), and Chip Wilson (Canada) – and Evotec’s CSO, Cord Dohrmann (Germany).