Drug Discovery

Whitehead Institute’s physician-scientists—researchers who also practice medicine—are highly motivated by the goal of improving patients’ outcomes. What drew them to Whitehead Institute is the possibility of making paradigm-shifting discoveries that can lead to major advances in medicine.

Chronic diseases such as diabetes are on the rise and are costly and challenging to treat. Whitehead Institute Member Richard Young and colleagues have discovered a common denominator driving these diverse diseases, which may prove to be a promising therapeutic target: proteolethargy, or reduced protein mobility, in the presence of oxidative stress.

Whitehead Fellow Aditya Raguram has received a 2024 NIH Director’s Early Independence Award. The prestigious funding award will support his work developing cell-derived bioparticles into safe and efficient vehicles for delivering therapeutic proteins into cells. 

Whitehead Institute Member Jonathan Weissman and collaborators have developed a gene-silencing tool that shows promise as a therapy against fatal prion diseases. The tool, a streamlined epigenetic editor, paves the way for a new class of genetic approaches to treat certain diseases.

The Institute's newest Whitehead Fellows are Valhalla Fellow Allison Hamilos and Aditya Raguram. Watch these videos to meet them and learn more about their work.

Researchers in Whitehead Institute Member Richard Young’s lab and colleagues at MIT show that a machine learning model can predict which subcellular compartments a drug will concentrate in based on its chemical features. This could be used to design safer and more effective drugs, and to understand how subcellular compartments govern diverse biochemical processes.

A pain-causing compound in the Australian stinging tree has applications for cancer treatment, but it’s difficult to harvest enough to study. Whitehead Institute scientists present a way to synthesize the compound in the leaves of harmless tobacco plants, and potentially in a culture dish.

Whitehead Institute Member Jing-Ke Weng studies plant metabolism, the set of processes plants use to produce thousands of unique molecules, many of which have potent medicinal properties. The Weng lab is hunting for more of these molecules in the wild, and developing strategies to sustainably produce plant molecules already of interest at scale.

Some of the most important tools in researchers’ toolkits are the model organisms they use to study biological questions. How do researchers decide which species, out of the millions that exist, to develop as models? Whitehead Institute researchers have had a hand in establishing and promoting the use of several model organisms over the years.