ASHEVILLE, North Carolina — Epidermolysis bullosa (EB), a heterogeneous congenital condition of skin fragility, received its first FDA-approved gene therapy only a few months ago, but accelerated progress across multiple treatment strategies predicts additional important and perhaps dramatic further progress, according to a prominent EB researcher.
Not only are recent developments in EB "exciting," the progress on multiple fronts for control of disease or its symptoms suggests "we are on the cusp of a new era," Jemima Mellerio, BSc, MD, a consultant dermatologist, St. John's Institute of Dermatology, London, England, said at the annual meeting of the Society for Pediatric Dermatology.
Published clinical studies of cell therapies and gene therapies date back at least 15 years, according to a review by Mellerio on why developments are starting to move so quickly. The difference now is that many obstacles to routine use of these options are being resolved, so that viable strategies have reached or are reaching phase 3 trials.
In addition to cell therapies and gene therapies, Mellerio discussed progress in three additional areas: gene editing, protein therapy, and drug repurposing.
Summarizing progress in each, she described improvement in levels of collagen VII, an important deficit in most types of EB, that were achieved with fibroblast injections that improved levels of collagen VII and anchoring fibrils in a
