A pointed approach to new medicine

Conventional drug development takes a scattershot approach. You uncover something going wrong with a particular gene and then create a compound to knock out that corresponding protein, regardless of its greater function within the cell. This approach is a bit like trying to understand a car by studying the steering wheel— without a complete picture of what’s happening between proteins, a drug can be less effective and come with unintended side effects.

Gandeeva Therapeutics uses a different method. They are trying to directly influence specific disease-driving protein interactions without affecting the rest of the protein functions. It’s the difference between using a floodlight and a laser pointer.

For decades, scientists have known that understanding the language by which proteins communicate is fundamental. Alterations in cellular protein structures and their interactions are implicated in nearly every disease.

Gandeeva starts with a rigorous identification of the optimal protein-protein complexes and surfaces to target. Then, by imaging these target interfaces at atomic resolution, they can make better decisions and, ultimately, better therapeutics.

It can take years and 3,000 to 6,000 compounds to go from the discovery stage to identifying a drug candidate for clinical trials. Through its highly collaborative and iterative weekly design cycle, Gandeeva can reduce this process to less than a year and by testing just a couple of hundred compounds.

How can they achieve this level of precision? Through cryo-electron microscopy (cryo-EM). Cryo-EM is a technique used in structural biology to study the 3D structures of macromolecules such as proteins. A protein is frozen in a thin layer of ice and placed into an electron microscope. Scientists take millions of images to generate a 3D model of the protein. With these detailed models, scientists can better understand the structure, movement, and function of specific proteins, including the interfaces at which proteins can bind together.

By deploying cryo-EM at every stage of the drug discovery process, Gandeeva can develop high-resolution models that allow them to identify promising molecules and to intelligently guide next steps. With the world’s most advanced imaging technology, Gandeeva is transforming lives with the next generation of medicine.