Share Public Profile
Stephen J. Pakola, M.D., is a seasoned physician executive with more than three decades of experience in the biopharmaceutical industry, including over 25 years serving as Chief Medical Officer. He has led global clinical development programs spanning small molecules, biologics, and gene therapies across multiple therapeutic areas, including ophthalmology, central nervous system disorders, and cardiovascular disease. Dr. Pakola is widely recognized for his leadership in advancing innovative therapies from early research through regulatory approval. He served as the lead inventor and program architect behind Jetrea®, the first globally approved pharmacologic treatment for vitreomacular adhesion. Throughout his career, he has played a central role in building and guiding high-performing clinical and regulatory teams, contributing to the successful development of multiple breakthrough therapies. Currently serving as Chief Medical Officer at REGENXBIO, Dr. Pakola continues to drive advancements in gene therapy, overseeing programs from investigational stages through pivotal trials and regulatory submissions. His work reflects a consistent commitment to scientific rigor, patient impact, and innovation in medicine.
