Establishing Biomarkers and Preclinical Proofof Concept in USH3Models

National Center For Advancing Translational Sciences

Investigators

Abstract

ased on the auditory proof of concept data, the lead collaborators have advanced BF844 into Phase I safety trials in healthy volunteers. To support further studies into the effects of BF844 on the visual phenotype, the NCATS team is launching the production of USH3 retinal organoids derived from patient cells. In parallel, metabolomic, transcriptomic and proteomic analyses of samples from the Phase I studies will help identify relevant biomarkers to support further clinical development along both phenotypic axes.