By Amy Bekier, San Diego, California
On July 30, 2016, twenty-five FSHD patients, family and scientists assembled at Genea Biocells in La Jolla, California, to hear presentations by senior scientists Amanda Rickard and Anabel de la Garza. Their work is aimed at identifying future treatments for FSHD patients.
The company’s website states that “Genea Biocells developed the world’s first human stem cell model of FSHD which shows cellular hallmarks of the disease. We pursue a small molecule approach to find therapeutic drug candidates that slow, stop or reverse the disease-related phenotype in skeletal muscle. This project has been supported by the FSHD Global Research Foundation and Friends of FSH Research.”
“Genea Biocells is a neuromuscular disease-focused discovery stage company using proprietary human pluripotent stem cell technologies. Genea Biocells also provides contract research services to pharma and supplies reagents to strategic academic collaborators to expand their capabilities and further validate their technologies. Genea Biocells has one of the world’s largest banks of pluripotent human embryonic stem cells and developed the world’s first consistent, scalable and high-yield differentiation process for functional skeletal muscle cells.”
Rickard offered a detailed and engaging explanation of how human embryonic stem cells have been developed into an “FSHD-in-a-dish” cellular model, and how this system is used to find potential therapies for FSHD. De la Garza presented a review of techniques for human muscle stem cell transplantation which could potentially present a customized treatment for each FSHD patient.
We all enjoyed a tour of the lab after a lunch hosted by the FSH Society. Thank you to Genea Biocells and especially to Amanda and Anabel for providing us with such a fascinating and educational afternoon!