Steve Pakola
Chief Medical Officer @REGENXBIO
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WORK HISTORY
Chief Medical Officer @REGENXBIO
Lead Medical Organization (Clinical Development/Operations, Drug Safety, Regulatory, Biometrics, Medical Affairs), built from 40 to 80 leaders strong, across ophthalmology and rare disease programs *Led clinical transformation from early-stage to late-stage company, with multiple programs advanced into pivotal clinical development • Design and execution of largest pivotal gene therapy clinical program ever. • Design and execution of first ever trials evaluating Gene Therapy delivery to novel space (suprachoroidal delivery for wet AMD and DR) • Advancement of Gene Therapy trials for treatment of serious, rare, monogenic diseases via CNS delivery (MPS-II and -1). • Translation of preclinical programs into clinical development (Successful IND filing for RGX-202 for Duchenne, and CTA for RGX-381 for CLN2 ocular)*Led Medical Organization component of Abbvie global strategic partnership for RGX-314 program ($370 million upfront with up to $1.38 billion in additional milestones)
EDUCATION
University of Pennsylvania
BA, MD
ABOUT STEVE PAKOLA
Industry Leader with over 25 years of biopharmaceutical experience, including over 20…
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