Expansion Therapeutics
Dr. Skerlj has more than 25 years of experience leading the discovery and development of disease modifying small molecule drugs to treat genetically defined rare diseases. He has authored 67 publications, holds 52 patents and has advanced multiple drug candidates into clinical development.
Most recently, Dr. Skerlj served as Chief Scientific Officer for X4 Pharmaceuticals, Inc., where he also was one of the company’s scientific founders and co-inventor of its lead drug candidate, mavorixafor. Prior to joining X4, Dr. Skerlj held leadership roles in drug discovery and development at Lysosomal Therapeutics where he co-invented LTI-291, a CNS drug in clinical development for Parkinson’s disease. Previous to that, he was interim Head of Small Molecule Discovery at Genzyme and part of the executive team at AnorMED, a publicly traded company that was acquired by Genzyme in 2006. Dr. Skerlj is an accomplished drug developer with two approved drugs; plerixafor, a CXCR4-targeted stem cell mobilizer approved by the U.S. Food and Drug Administration (FDA) in 2008, and ertapenem, an anti-bacterial approved by the FDA in 2001. Dr. Skerlj received his Ph.D. in synthetic organic chemistry from the University of British Columbia, and completed postdoctoral fellowships at the University of Oxford and Ohio State University.
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Expansion Therapeutics
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Expansion Therapeutics is a drug discovery and development company pursuing the vast potential of small molecule medicines for RNA-mediated diseases. Expansion has assembled the intellectual property, know-how, and proprietary enabling technologies and tools necessary to facilitate the creation of potent and specific small molecule binders of RNA. Through this unique platform, Expansion is building a portfolio of novel RNA-targeted drug candidates with activity across a broad number of disease indications. The company’s initial development focus is on therapies for patients with expansion repeat diseases who currently have limited and unsatisfactory treatment options. Expansion is based in San Diego, California and Jupiter, Florida.