Three entrepreneurs who have been affected personally by FSHD announced at this year’s FSHD Champions meeting that they have established a biotech dedicated to developing a treatment for FSHD. The new company, called Facio Therapies, will develop a small molecule that boosts the activity of the enzyme SMCHD1. Mutations in the SMCHD1 gene have been linked to FSHD2, and reduced SMCHD1 activity results in an increased likelihood of the expression of DUX4, a gene implicated in FSHD.
Facio states on its website that “Our therapy will be affordable and made available to as many people with FSHD as possible. We will develop our therapy as quickly and as cost-effectively as possible, using innovative ways to cut the duration and cost of drug development. Along the way, we will publish the results of every study we sponsor.”