Staff Profile: Juan Marugan

Juan Jose Marugan, Ph.D.
Juan Jose Marugan, Ph.D.

Leader, Chemistry

Division of Pre-Clinical Innovation

National Center for Advancing Translational Sciences

National Institutes of Health

Email Juan Jose Marugan


Juan Marugan has more than 20 years of experience in drug discovery and development, from classical medicinal chemistry to structure-based drug design, high-throughput screening, enzymatic and cell assays, and in vivo models. His involvement in pharmaceuticals began during his doctoral studies, carried out in in partnership with Pharma Mar S.A., a Spanish pharmaceutical company involved in the development of marine natural products. After holding medicinal chemistry postdoctoral positions at the University of Pittsburgh and Tufts University, Marugan moved to the pharmaceutical sector, holding positions of increasing responsibility from research scientist to head of pre-clinical drug research.

Marugan joined NIH in 2008 and subsequently joined NCATS. He has extensive experience as a team leader of programs at the lead optimization stage; several leads have been licensed to pharmaceutical companies, and several molecules have advanced toward pre-clinical development or clinical trials. Marugan’s job responsibilities have included lead discovery and optimization with early absorption, distribution, metabolism and excretion as well as pharmacokinetics profiling; managing internal and external resources; structure-activity relationship optimization; chiral separation; and in vivo studies.

Marugan holds a Ph.D. in organic chemistry from the Universidad Complutense de Madrid and a B.S. in chemistry from the Universidad de Salamanca.

Research Topics

During the course of his scientific life, Marugan has been involved in the investigation of active compounds in numerous therapeutic areas, including antivirals; antibiotics; oncology; and cardiovascular, central nervous system, and rare and neglected diseases. He serves as a co-leader of an interdisciplinary project team with a diverse target portfolio, including:

  • Lysosomal storage disorders: Niemann-Pick disease type C, Gaucher disease, Pompe disease, metachromatic leukodystrophy
  • Neurodegenerative disorders: Alzheimer’s disease, Huntington’s disease, amyotrophic lateral sclerosis, Parkinson’s disease, spinal muscular atrophy
  • Neurodevelopment and mood disorders: autism, neurogenesis and synaptogenesis, schizophrenia
  • Cancer: metastasis, immune activation, Eya phosphatase
  • G protein–coupled receptor modulators: RXFP1, GPR120, GPR110, GPR32, TSHR, D2R
  • Virus: influenza, hepatitis C virus, Venezuelan equine encephalitis virus

Selected Publications

  1. Pharmacokinetic evaluation of the PNC disassembler metarrestin in wild-type and Pdx1-Cre;LSL-KrasG12D/+;Tp53R172H/+ (KPC) mice, a genetically engineered model of pancreatic cancer.
  2. Neural stem cells for disease modeling and evaluation of therapeutics for Tay-Sachs disease.
  3. Optimization of the first small-molecule relaxin/insulin-like family peptide receptor (RXFP1) agonists: Activation results in an antifibrotic gene expression profile.
  4. A widely-applicable high-throughput cellular thermal shift assay (CETSA) using split Nano Luciferase.
  5. High-throughput screening identified selective inhibitors of exosome biogenesis and secretion: A drug repurposing strategy for advanced cancer.