Gene Therapy

Our last gene therapy article highlighted several novel cures for rare, life-threatening illnesses using gene therapy technology. Many more treatments based on gene therapy are in the clinical trial phase. Despite the remarkable results thus far, significant challenges lie ahead. As with any new treatment modality, scientists must be cautious in their approach, carefully studying the potential side effects in the short and long term. Unlike conventional treatments, gene therapy research raises unique economic, distribution, and manufacturing concerns. Below, we will discuss the medical and social hurdles posed by gene therapy research and commercialization, and the nascent regulatory response underway to help move this new frontier of medicine forward in a safe and ethical manner.

It is no surprise that one of the most impactful health stories of 2023, as reported by Scientific American, is gene therapy, specifically the approval by the U.S. Food and Drug Administration (FDA) of a groundbreaking gene therapy treatment for sickle cell disease using a novel genome editing technology called CRISPR.