Drugging the Undruggable

Explore how we’re addressing long-standing challenges in drug discovery.

Modern medicines can unmask cancer cells hiding from our immune systems, hunt down the rogue immune cells that underlie multiple sclerosis, and pull proteins together to drive the blood clotting process in people living with hemophilia.

And yet, for all the progress that’s been made, there are still thousands of molecules in our bodies that are known to influence disease but are beyond our ability to affect. In fact, it’s estimated that out of roughly 4,000 disease-related targets known to us today, only a quarter actually have a medicine that can reach and act on them. Researchers sometimes call these elusive targets “undruggable.”

But we don’t like to use that term. It makes the task sound impossible—and we don’t believe it is.

“We have drugged the ‘undruggable,’ and we will continue to do so,” says Wendy Young, Senior Vice President, Small Molecule Drug Discovery. “What we’re really talking about is hitting even more of these tougher targets routinely.”

We’re employing an interdisciplinary scientific arsenal of approaches that we hope will give us access to more of these previously inaccessible targets. A lot of these tools help us to explore entirely new treatment approaches, like T-cell therapy—an emerging type of treatment that removes a person’s immune cells, programs them for a specified task and then reinfuses them back into the body—or individualized cancer vaccines, which are uniquely tailored to the genetic makeup of an individual’s tumor.

“It’s an exciting time because we’re moving beyond our normal programs and familiar modes of treatment,” says Young. “As a company we’ve always challenged ourselves to break new ground, and it’s inspiring to continue to explore uncharted territories.”

Often the best way to explore is through partnerships with other companies or institutions that allow us to pool our expertise. For example, we’ve partnered with Adaptive Biotechnologies on neoantigen T-cell therapies, and with Skyhawk Therapeutics to target genetic causes of cancer and neurodegeneration. We see partnerships like these as a crucial component of expediting progress in drug discovery.

“We emphasize disease areas with high unmet needs like neuroscience, oncology, immunology and ophthalmology,” says Tapan Maniar, Site Head for Global Research Technologies, Pharma Partnering. “When we find a promising technology or modality platform with the potential to tackle important, but challenging targets in those disease areas, we go after it.”

The reality is that not every new approach leads to a breakthrough medicine. And we openly embrace this as part of the scientific process. By expanding our knowledge and ability to manipulate biology at the molecular level, we continue to drive scientific breakthroughs and bring new and better medicines to patients.



Keep your eye on the Drugging the Undruggable section of gene.com as we highlight our latest approaches to tackle long-standing challenges in drug discovery.