SAB Member

Dr. Renato Skerlj has 30 years of pharmaceutical experience in the discovery and development of small molecule drugs to treat cancer, infection, immune mediated and rare diseases. He is an accomplished “drug hunter’ with two marketed drugs and multiple drugs in clinical development. These include Invanz®, an anti-infective approved in 2001 and Mozobil®, a stem cell mobilizer approved in 2008. He is currently the CEO of Expansion Therapeutics, a company developing transformative small molecule medicines for RNA-mediated diseases. Previously he was the CSO and founder of X4 Pharmaceuticals, responsible for the development of Mavorixafor which is in Phase 3 studies for WHIM syndrome as well as earlier studies for Waldenstrom’s Macroglobulinemia. Prior to that he was the SVP of Drug Discovery and Development at Lysosomal Therapeutics where he invented and developed LTI-291 for GBA Parkinson’s disease currently entering Phase 2 development. Earlier he was the interim Head of Small Molecule Discovery at Genzyme and VP of Medicinal Chemistry where his group was responsible for identifying Venglustat which is in numerous late-stage clinical trials. Prior to joining Genzyme, Dr. Skerlj was part of the executive team at AnorMED, a publicly traded company acquired by Genzyme for $580M in 2006. He was an inventor and played a key role in the successful development and regulatory approvals of Mozobil®. He started his career with research positions at Merck and Johnson Matthey. Dr. Skerlj is the inventor of 52 patents, including those covering Invanz, Mozobil, Mavorixafor, Venglustat and LTI-291 and is author on 70 scientific publications. He is a member of the SAB of Vasomune Therapeutics and Kera Therapeutics. Dr. Skerlj earned his PhD in synthetic organic chemistry at the University of British Columbia and completed post-doctoral fellowships at the University of Oxford and at Ohio State University.