Staff Profile: Wei Zheng

Wei Zheng, Ph.D.
Wei Zheng, Ph.D.

Leader (Biology)

Division of Preclinical Innovation
Therapeutics for Rare and Neglected Diseases

National Center for Advancing Translational Sciences

National Institutes of Health

Email Wei Zheng


Wei Zheng leads a group of biologists in support of preclinical drug development as part of the Therapeutics for Rare and Neglected Diseases (TRND) program. His lab focuses on advancing TRND projects through preclinical drug development to Investigational New Drug application and clinical trials. In addition, he 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.

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.

Zheng received his doctorate 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.

Research Topics

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. Zheng also serves as editor-in-chief of the peer-reviewed journal Current Chemical Genomics and Translational Medicine.

Selected Publications

  1. PKM2 inhibition may reverse therapeutic resistance to transarterial chemoembolization in hepatocellular carcinoma
  2. An induced pluripotent stem cell line (TRNDi001-D) from a Niemann-Pick disease type C1 (NPC1) patient carrying a homozygous p. I1061T (c. 3182T>C) mutation in the NPC1 gene
  3. Phosphocyclocreatine is the dominant form of cyclocreatine in control and creatine transporter deficiency patient fibroblasts
  4. A cell-based, infectious-free, platform to identify inhibitors of lassa virus ribonucleoprotein (vRNP) activity
  5. 17-Hydroxy Wortmannin Restores TRAIL's Response by Ameliorating Increased Beclin 1 Level and Autophagy Function in TRAIL-Resistant Colon Cancer Cells