The Critical Role of Patient Community-Industry Partnership in Advancing a Gene Therapy

Friedreich’s ataxia is a progressive, multisystem disease that robs people of coordination, independence, and often life itself. Until recently, there had been no approved therapies. In Friedreich’s ataxia, a genetic mutation causes a deficiency in frataxin, a protein that plays an essential role within mitochondria and affects enzymes involved in energy production. Solid Biosciences is developing a gene therapy designed to restore frataxin where it is needed. Friedreich’s Ataxia Research Alliance CEO Jennifer Farmer and Solid Biosciences chief medical officer Gabriel Brooks discuss the lived reality of Friedreich’s ataxia, Solid Bioscience’s next-generation gene therapy now in development to address the underlying cause of the disorder, and what genuine partnerships between patient organizations and industry look like when patient priorities drive trial design and development decisions.