Christopher Evans, PhD, Mayo Clinic, Rochester, MN, provides an overview of research into healing bone defects using gene therapy. Bone grafts such as Infuse, which contains recombinant human bone morphogenetic protein-2 (rhBMP-2), can have severe side effects and is expensive due to challenges with delivery, meaning a high dose is required. Gene therapy approaches offer the potential to overcome these challenges, such as the use of adenovirus vectors to improve delivery of BMP-2. The transfection of chemically modified mRNA encoding BMP-2 protein is also being investigated and has proven to be efficacious in healing bone defects in preclinical models. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.
Christopher Evans is co-founder of Genascence Inc. that is seeking to commercialize our gene therapy for osteoarthritis.