By the time a new treatment reaches a patient, it has already passed through years—often decades—of scientific testing, data analysis, and regulatory review. What may look like a simple decision in a doctor’s office reflects an intricate process that few people outside the industry ever see.
Few understand that process better than Dr. Steve Pakola, a physician who has spent his career navigating the intersection of science, medicine, and product development.
Pakola began his career as a clinician but soon recognized a broader path to impact.
“During my training, I realized that as a physician, you can help individuals directly—but through research and development, you can also create treatments that benefit many more people,” he said.
That insight set the direction for a career spanning more than three decades, including over 25 years as a chief medical officer. His work has ranged across therapeutic areas such as ophthalmology, cardiovascular medicine, and rare diseases, encompassing small molecules, biologics, and gene therapies.
1. From Concept to Clinic
Since 2019, Pakola has served as chief medical officer at REGENXBIO, a company developing gene therapies for rare and inherited diseases. His focus is on shepherding them through the intricate pipeline of testing, manufacturing, and regulation needed to make them real.
Among the late-stage programs under his watch are RGX-314 for eye diseases wet AMD and diabetic retinopathy, RGX-121 for the rare condition mucopolysaccharidosis II, and RGX-202, in development for Duchenne muscular dystrophy.
Progress at this level is less about eureka moments than about coordination and execution. “It’s not just the science,” Pakola said. “It’s about keeping every piece — research, operations, manufacturing, and regulation — moving together.”
2. Lessons in the System
Before becoming a senior leader, Pakola learned drug development from the inside out — running, analyzing, and defending clinical trials. Early in his career, at Quintiles, he helped run cardiovascular studies through all trial phases as well as presenting at an FDA Advisory Committee mtg. At Organon and Boehringer Ingelheim, he contributed to development programs that faced the scrutiny of regulatory agencies, building an understanding of what separates promising drugs from approvable ones.
Later roles at ThromboGenics, Amakem, and Aerpio Therapeutics expanded his vantage point — from data to strategy, from molecules to entire portfolios. Each phase revealed a different part of the system: how science meets infrastructure, and how timing, design, and communication can shape whether a treatment ever reaches patients.
A Case Study in Translational Medicine: Ocriplasmin
One of Pakola’s most defining projects offers a window into how discovery becomes medicine. At ThromboGenics, he introduced and led the development of ocriplasmin (Jetrea®), resulting in product approval in the U.S. and Europe as the first pharmacologic treatment for vitreomacular adhesion including macular hole.
The process took years and repeated reinvention. Originally studied in the context of stroke, Pakola came up with the idea to repurpose the molecule to potentially treat vision threatening eye disease. Steering that transition meant building a clinical and regulatory path from scratch — trial design, global submissions, and market preparation — while the company itself grew from a small research team into a public biotech.
The experience solidified his view of drug development as not only a scientific challenge but a logistical and strategic one—how to turn discovery into accessible treatment.
3. Early Lessons in Gene Therapy
Pakola’s first exposure to gene therapy came at the University of Pennsylvania, where he completed both undergraduate and medical degrees and worked in the lab of Dr. Jean Bennett — a pioneer in retinal gene therapy. His research contributed to early preclinical studies using adeno-associated virus (AAV) vectors.
At the time, gene therapy was considered experimental. Decades later, many of those foundational concepts are starting to pay off. For Pakola, now back in the field at a more mature stage of its evolution, the experience feels full circle: “The science is catching up to the vision,” he said. In fact, under Pakola’s leadership, REGENXBIO is now running the largest gene therapy clinical program ever conducted (RGX-314 for treatment of wet AMD).
4. The Perspective Behind the Work
Over the years, Pakola has received recognition for his work, including the Summit of Excellence Lifetime Achievement Award for contributions to treatments for retinal disease. He has published extensively across multiple therapeutic areas, and sits on the Gene Therapy Advisory Group within the Alliance for Regenerative Medicine.
Reflecting on his career, Pakola emphasizes perseverance and process. He said “Over 90% of drugs that enter development fail. You have to learn how to get back on your feet and keep moving forward. So learn from and enjoy the process as much as the goal.”
For Pakola, success lies both in delivering new therapies to patients and families as well as impacting people along the way.
