Inside Vincere’s quest to transform treatments against neurodegeneration using artificial intelligence
What happens when a software engineer marries a neuroscientist? In Andy Lee and Dr. Spring Behrouz’s case, it sparked dinner-table conversations that grew into a groundbreaking quest—not just to tackle Parkinson’s disease, but to take on aging itself.
“My wife was seeing vast amounts of human data going unused in her neuroscience work,” Andy recalled, “while I was starting to play with high-performance computing. We decided to combine our expertise to see if we could predict what would stop Parkinson’s disease.”
It was a bold endeavor. Parkinson’s disease—and neurodegeneration in general—represents one of medicine’s greatest challenges. “Everything that has been tried has failed,” Andy noted, “and yet the puzzle pieces seem to be emerging. We believed AI could put them together in a way humans alone couldn’t.”
Following the data to an unexpected target
Soon after, Vincere Biosciences was born—a precision medicine company based in Boston, Massachusetts that’s focused on advancing small molecule therapeutics. Its AI platform sifted through vast troves of biological data, connecting dots no human researcher had yet seen. The trail led to an unexpected suspect: damaged mitochondria, the cellular powerhouses responsible for generating energy in every cell.
The team developed molecules that could turbocharge the process of repairing these damaged mitochondria. But it was their specific target that proved years ahead of the field. In 2018, Vincere’s platform pinpointed a little-known enzyme, USP30, as a critical pathway for Parkinson’s—long before it was on anyone else’s radar. At the time, only 18 published papers mentioned it in the scientific literature, and just one linked it to Parkinson’s disease.
This was exactly the kind of emerging target Vincere was looking for. “If you have a completely novel target no one is looking at, you might never get the support you need,” Andy reflected. “If you chase what everyone is already chasing, that’s also problematic because you aren’t contributing much. But if you find a new thread that can unravel the whole disease—those are the breakthroughs worth pursuing.”
The power of virtual cells
To validate their discovery, the Vincere team built “virtual cells”—sophisticated models that simulate biological processes. They started by using healthy human brain cell data from the Allen Institute for Brain Science, building digital replicas of human brain cells and predicting what would change with a genetic mutation that causes Parkinson’s or in a cell with sporadic Parkinson’s. These simulations predicted that inhibiting USP30 could reverse dysfunction for a large proportion of Parkinson’s patients.
The Michael J. Fox Foundation recognized the potential early, taking a bet on an unproven platform with a daring idea to simulate biology by funding to validate the simulations against preclinical models. What convinced them to support such an early-stage concept? According to Andy, it was Vincere’s clear focus on patient impact. “Even though we were unproven, we showed exactly how our work could accelerate a cure,” he explained. It was an audacious pursuit, but MJFF-funded collaborations with Mayo Clinic and University of Oxford validated about three-quarters of their AI predictions against lab experiments. That early support paved the way for Vincere to raise additional capital from outside investors.
The scientific community has since dramatically validated Vincere’s early insight. In the seven years since Vincere nominated USP30 as their target, the evidence has mounted. Now, there are close to 100 papers that have shown USP30 reduction being protective in a variety of different conditions. This validation reached a pinnacle last year when the Michael J. Fox Foundation conducted a comprehensive academic collaboration to identify targets that would stop Parkinson’s, naming USP30 among the top five.
Looking beyond Parkinson’s
Vincere’s mission has expanded from a quest to stop Parkinson’s into something potentially transformative for human aging. Since mitochondrial dysfunction underlies many age-related conditions, the company’s compound shows promise beyond neurodegeneration. What sets Vincere’s approach apart is its precision. Unlike existing drugs that work by introducing damage—making them neither particularly safe nor potent—Vincere’s drugs use a much more targeted approach.
“In laboratory models—flies, worms and mice—boosting this mechanism can extend lifespan by up to 30%,” Andy explained. “While it’s too early to promise the same in humans, the potential impact on healthy aging could be enormous.”
This is where strategic partnerships become essential. While Vincere excels at computational discovery and early medicinal chemistry, translating breakthrough science into human therapeutics requires specialized expertise in preclinical development and regulatory navigation. KreaMedica brings exactly this expertise to the table.
“Everyone is fully focused right now on getting our Parkinson’s molecule into the clinic,” Andy said, describing the intensive toxicology safety testing that will give regulators and patients confidence that the drug can be taken without harm. “KreaMedica is helping to refine our plan and oversee the work to ensure that our molecule can successfully make that important jump from animal studies to human studies.”
A vision for the future
Looking beyond the immediate clinical trials, Andy envisions a future where Vincere’s therapy addresses not only Parkinson’s but a broad range of age-related diseases. “Given the mechanism and the way mitochondria are at the core of aging and neurodegeneration, we see this as an exciting time. After clinical trials, we could begin testing in other areas,” he said. The ultimate goal: a widely available therapeutic that slows the rate of aging—delaying cardiovascular disease, kidney disease, Alzheimer’s and other conditions.
This ambition is powered by what Andy calls an unprecedented moment for computational drug discovery. “You can think about AI as a big compression of knowledge and reasoning. All of this knowledge is right there at your fingertips, instantly accessible,” he explained. “Everyone needs to be looking at how to incorporate AI into every aspect of drug development. There isn’t a single stage in the discovery-to-clinic pipeline that can’t be accelerated with this technology—if it’s used right.”
Vincere’s journey demonstrates that when bold computational discovery meets the right partners, world-changing science advances. With their AI-discovered target now validated by the scientific community and KreaMedica guiding the preclinical path, Vincere is poised to take the next crucial step in bringing hope to millions affected by Parkinson’s and age-related diseases.
For more information about investment opportunities in Vincere—or to learn how KreaMedica can accelerate your biotech venture—contact us.