The innate immune system is full of targets that could be harnessed against diseases from cancer to lupus to epilepsy. But researchers have struggled to hit them because they didn’t know the structure of those targets.
“We didn’t know the confirmation [of the targets], where the pockets are, how big they are,” Marcelo Bigal, CEO of Ventus Therapeutics, told FierceBiotech. “Which means trying to develop a drug against something you don’t see—it’s drug development with the lights off.”
With its structural immunology technology, Ventus is switching the lights on, emerging from stealth with $60 million from Versant Ventures and GV to advance three programs. The company is developing multiple small molecules against each of the three targets but is keeping specific disease areas under wraps, Bigal said.
The company is working to inhibit the targets, which could be useful in several areas including brain, immune and cardiometabolic disorders as well as monogenic diseases, in which a genetic mutation causes the body to make too much of a disease-causing protein. But it’s also looking to stimulate those targets, which could come in handy fighting cancer.
Ventus’ pipeline aims at two main pathways of the innate immune system: the inflammasome and the DNA-sensing pathway, also called the interferon pathway. They form the body’s first line of defense, sensing danger within cells and “sacrificing” those cells to contain the threat, Bigal said. The company’s platform does more than elucidate protein structure—it also surmounts problems scientists have faced when trying to drug the targets in a lab dish.
The targets exist as monomers, or small pieces of proteins, inside cells until they sense a threat. In response, they’ll assemble into a larger protein, Bigal said.
“But when you put monomers in vitro to do drug development, they immediately oligomerize and become this very large chunk of protein that nobody can handle,” he said. With Ventus’ technology, its scientists can purify and immobilize these proteins, so they can study drugs in vitro before moving on to animal studies, where in the past, they would have gone straight to animal studies in a trial-and-error approach.
Ventus’ three programs are fully funded through “meaningful inflection points,” but Ventus has nine other targets that it’s not actively working on, Bigal said. All 12 offer partnership opportunities, whether a partner is interested in pursuing its three selected targets in other disease areas or in a target-based, disease area-based or platform-wide partnership.
“Because we are well-funded, that means in the next month, it’s pipeline, pipeline, pipeline … But meanwhile, we are allowing ourselves to have very selective conversations with pharma to foster possibilities for a quality partnership,” Bigal said.
The company, which is based in Boston and Montreal, plans to grow to more than 30 scientists this year.