Spotlight on R&D: From Molecules to Medicines
Thinking back on the path I took getting into drug discovery, I remember my childhood curiosity about the ways that science explains the workings of the world. I eventually became fascinated with the rules that allow chemical structures to fit together like puzzle pieces, allowing us to build new molecules. During my high school and undergraduate education, I was deeply fortunate to have had wonderful mentors who helped me develop a focus on organic chemistry. During my graduate studies I began to conceptualize medicinal chemistry as an intricate application of organic chemistry concepts to probe biological systems. The ability to use chemistry to ask new questions about biology really connected with me and eventually led me to work in drug discovery. Being able to marry my scientific interests with the opportunity to have an impact on human health has provided powerful motivation throughout my career.
Today, as Director of Medicinal Chemistry at Variant Bio, I am leading our small molecule drug discovery programs. My academic training focused on synthetic organic chemistry, so designing and creating novel molecules is at the core of what I do. Those new molecules aren’t especially interesting on their own, though, so medicinal chemists work extensively with colleagues from other areas of R&D to understand what purpose our molecules could ultimately serve.
I joined Variant Bio to help build up our chemistry capabilities, which involves coordinating both internal and external teams to meet the evolving needs of our discovery pipeline. Sam Parker also recently joined the team as Senior Research Associate and has helped to get our chemistry lab fully established. She held positions at a couple of small biotech companies prior to joining Variant Bio, so she has experience working with a lean, highly collaborative drug discovery team. Sam works in the lab synthesizing new compounds — that encompasses making compounds to test in our drug discovery assays, as well as devising new methods to create novel molecules. Like most small biotech companies, Variant Bio also contracts with external teams in North America, Europe, and Asia to augment our capabilities and accelerate project progress. Sam’s chemistry will dovetail with the work done through these extended teams, ultimately allowing Variant Bio to generate molecules that can answer key questions about our biological targets.
Once Variant Bio’s genetic insights have been validated as targets for therapeutic intervention, the chemistry group begins work toward discovering compounds that can act on the biological pathways. A new molecule needs to meet many criteria before it can enter human clinical trials. The genetic data underpinning our drug discovery projects can help us refine different parts of a lead molecule so that its profile is tailored to the specific therapeutic need.
Variant Bio’s chemistry team works with members of the biology group in different ways. We often help to identify molecules from the literature that can be used as tools to help validate new targets identified from our genetic platform. This can include testing compounds in cellular assays to understand disease biology, as well as animal models of certain diseases to help us translate genetic insights into drug discovery programs. Once a medicinal chemistry project is underway, the chemists constantly interact with biologists and DMPK scientists to advance new molecules through assays of increasing complexity toward clinical development.
Drug discovery involves many different disciplines all working together to forge a path toward a compound that can be viable for use in human studies. Together, we have the opportunity to discover new medicines in really unique ways. I embrace the challenge of coordinating those moving parts with urgency to deliver molecules that will be safe and effective in clinical trials.