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Amanda Rickard--someone well known to the FSHD community as one of those exceptional scientists who love to help patients and families understand what's going on at the frontiers of FSHD research.
Amanda Rickard is a scientist focused on FSHD therapeutics development at Genea Biocells. She began her work on FSHD in Dr. Dan Miller’s lab at the University of Washington’s Institute for Stem Cell and Regenerative Medicine in Seattle. Three years ago she moved to San Diego to lead Biocells’ efforts toward identifying a disease-modifying treatment for FSHD patients. She also serves as an FSH Society volunteer, participates in patient community groups, and sits on the Board of Directors and Scientific Advisory Board for Friends of FSH Research.
Genea Biocells is an early stage drug development company focused on using stem cell technologies to identify treatments for muscle diseases. After generating a series of FSHD-affected human embryonic stem cell lines (supported by FSHD Global), Biocells developed a method for generating muscle cells from stem cells in a dish, yielding an unlimited source of FSHD-affected muscle cells for drug screening (supported by Friends of FSH Research). The group has since identified drug candidates that silence DUX4 expression in FSHD muscle, including GBC0905, which received FDA Orphan Drug Designation in 2018 for the treatment of FSHD. Amanda and the Genea Biocells team are now working to complete the studies necessary to bring GBC0905 to clinical trials.