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Staff Profile: Wei Zheng

Wei Zheng, Ph.D.

Leader, Biology, Therapeutics for Rare and Neglected Diseases

Division of Pre-Clinical Innovation

National Center for Advancing Translational Sciences

National Institutes of Health

E-mail Wei Zheng


Wei Zheng received his Ph.D. in pharmacology from the State University of New York at Buffalo, where he studied ion channel pharmacology and completed a postdoctoral fellowship in molecular biology. Prior to joining NIH in 2005, Zheng spent 12 years as a researcher at pharmaceutical companies, including Berlex, Amgen and Merck, where he gained broad experience in drug target and lead discovery in cancer and in cardiovascular, autoimmune, neurodegenerative and infectious diseases.

At NIH, Zheng has collaborated on high-throughput screening and probe discovery projects that led to identification of more than two dozen small molecule probes for use as research tools. He also has conducted drug discovery and development research for rare and neglected diseases. Currently, he is leading a group of biologists to support pre-clinical drug development as part of the Therapeutics for Rare and Neglected Diseases (TRND) program. Zheng also serves as editor-in-chief of the peer-reviewed journal Current Chemical Genomics and Translational Medicine.

Research Topics

Zheng’s lab focuses on advancing TRND projects through pre-clinical drug development to Investigational New Drug application and clinical trials. In addition, Zheng collaborates with researchers from NIH and academia on drug repurposing screens to accelerate drug development for rare and neglected diseases, focusing on lysosomal storage diseases and infectious diseases. Zheng’s lab also uses patient-derived induced pluripotent stem cells to develop new cell-based disease models, which can be used to evaluate drug efficacy.

Selected Publications

  1. Quantitative high-throughput screening identifies cytoprotective molecules that enhance SUMO conjugation via the inhibition of SUMO-specific protease (SENP)2.
  2. Identification of 4-phenylquinolin-2(1H)-one as a specific allosteric inhibitor of Akt.
  3. Repurposing a novel parathyroid hormone analogue to treat hypoparathyroidism.
  4. Drug discovery and development for rare genetic disorders.
  5. Drug repurposing screens and synergistic drug-combinations for infectious diseases.