Update: Determining in vitro and in vivo efficacy of novel DUX4 inhibitors

Posted by Friends of FSH Research on Apr 6, 2023

Update by Dr. Ozes
See also Determining in vitro and in vivo efficacy of novel DUX4 inhibitors

We are honored to be supported by Friends of FSH to test our direct DUX4 inhibitors in vivo to find a therapy to safely and effectively treat FSHD patients.

Using the funds from Friends of FSH Research, we have developed additional small molecule inhibitors against DUX4 and determined their drug-like properties. We demonstrated multiple compounds with enhanced anti-DUX4 activity in FSHD patient cells, in addition to increasing solubility and muscle exposure in mouse models. Significantly, we have demonstrated that Altay's DUX4 inhibitors are orally bioavailable enabling us to deliver our therapies to FSHD patients in tablet form.

We have since added to this research and show significant in vivo efficacy of our DUX4 inhibitors in a humanized animal model.

See Altay Therapeutics - Science 101 video on technology overview.