FSHD Phase I/II clinical trial previews for Scholar Rock, Soufflé and Dyne.

"If you want something bad, you have to fight for it. Step up you game, Scott. Break out the L-word". Today we discuss a little more on the Epicrispr clinical trial and preview the upcoming Scholar Rock, Soufflé and Dyne phase I/II clinical trials. As you know, the Devil is in the details. We are tingling with anticipation for the upcoming clinical trial report from Epicrispr to be presented at the World Muscle Society first week of October (DNA methylation and DUX4 target gene expression data from biopsies, please). We are still very hopeful for the Avidity Phase 3 trial currently in 45 sites around the world! Sure the DM1 trial tanked, but their DMD trial is progressing well. Regardless, new therapeutic approaches are here. We are very high on Soufflé's cell specific targeting platform as well as Dyne's alternate transferrin receptor targeting, both of which we hope will allow for increased dosing, more DUX4 knockdown, and greater intervals between dosing. We are also cautiously optimistic about Scholar Rock entering the FSHD space coming off of their recent FDA approval for a combined therapy in SMA. Truly exciting times (and more to come!), and we will try to keep you all updated on the science.