Chief Medical Officer
# Steve Pakola, MD
Steve Pakola, MD, is the Senior Vice President and Chief Medical Officer at REGENXBIO Inc. (RGNX), where he oversees clinical programs including gene therapies like RGX-111 for Mucopolysaccharidosis Type I (MPS I), RGX-202 for Duchenne muscular dystrophy, and RGX-314 (survevec) for wet AMD and diabetic retinopathy in partnership with AbbVie.[[1]](https://ois.net/steve-pakola-md/)[[2]](https://www.prnewswire.com/news-releases/stephen-pakola-oversaw-rgx-111-safety-claims-that-cost-rgnx-investors-millions-suewallst-302712044.html)[[4]](https://www.youtube.com/watch?v=prJebeIEqX0)[[5]](https://www.youtube.com/watch?v=hu3y4WUtIIU) With over 20 years in biopharmaceuticals, he has led preclinical and clinical development, regulatory affairs, focusing on retinal disorders such as diabetic retinopathy, diabetic macular edema, and age-related macular degeneration, as well as non-ophthalmic diseases.[[1]](https://ois.net/steve-pakola-md/) He recently sold 5,124 shares of RGNX stock on March 10, 2026, for $53,033 under a Rule 10b5-1 plan, retaining 247,926 shares.[[3]](https://www.investing.com/news/insider-trading-news/pakola-steve-regenxbio-cmo-sells-53k-in-rgnx-stock-93CH-4558616)
Dr. Pakola was the lead inventor and program lead for Jetrea® (ocriplasmin) from 2002 to 2012, achieving US and European approvals, and previously served as Chief Medical Officer at Amakem NV and ThromboGenics Inc., and in senior roles at Aerpio, Boehringer Ingelheim (Global Medical Lead for lipid-lowering and Dabigatran programs), Quintiles Cardiovascular Therapeutics, and Organon.[[1]](https://ois.net/steve-pakola-md/) He earned a B.A. summa cum laude and M.D. from the University of Pennsylvania, elected to the Alpha Omega Alpha Honor Medical Society.[[1]](https://ois.net/steve-pakola-md/) In 2026, he faces scrutiny in a securities class action over RGX-111 safety statements.[[2]](https://www.prnewswire.com/news-releases/stephen-pakola-oversaw-rgx-111-safety-claims-that-cost-rgnx-investors-millions-suewallst-302712044.html)
View full insider profile →
Discussion