Bexorg: Transforming CNS Drug Discovery with Whole-Brain AI

Funding announcements in biotech are more than business headlines—they're signals of scientific momentum and bets on new ways of understanding or treating disease. These moments offer a glimpse into where science is headed, what investors are prioritizing, and how novel biology is making its way toward the clinic. 

Next up: Bexorg, a pioneering company that's redefining how researchers approach central nervous system drug development.​

Bexorg's Bold Goal: Address the CNS Failure Crisis

Bexorg, a New Haven, Connecticut-based biotech company spun out of Yale University research, is emerging with a clear focus: to transform central nervous system (CNS) drug discovery through the world's first integrated AI and whole-human brain platform. The company was founded by Dr. Zvonimir Vrselja and Dr. Nenad Sestan, Yale scientists who developed groundbreaking technology to restore metabolic activity in postmortem brains.​

The challenge Bexorg addresses is staggering—industry reports have found that the clinical trial failure rate in CNS drug development exceeds 95%, largely because cellular and animal models fail to capture the extraordinary complexity of human brain biology. For context, before 2021, the FDA had not approved a new Alzheimer's treatment in 18 years.​

Bexorg is shifting this paradigm with its platform based on comprehensive human datasets generated from wet-lab experimentation in whole human brains. The company aims to address the translational gap that causes most CNS drugs to fail by enabling testing directly in human brain tissue from day one.​

Platform Technology & Capabilities

At the core of Bexorg's innovation is BrainEx, a sophisticated perfusion system that uses artificial blood to restore metabolic and molecular activity in whole, isolated human and pig brains. This technology allows researchers to study intact, physiologically active tissue that closely mirrors how drugs might act in living systems.​

The platform works by connecting donated brains to a system of tubes that perfuse the organ with a solution designed to mimic human blood. These lines are analogous to veins and blood vessels, perfusing the brain with artificial blood that can carry drug compounds for testing. BrainEx restores certain molecular and metabolic functions in postmortem human brains, creating what researchers call a functionally active system of diseased and non-diseased whole brains that enables unprecedented insights.​

Researchers obtain both diseased and healthy brains through established organ-donation networks, allowing them to compare responses side-by-side. The company can extract high-resolution transcriptomic, proteomic, and metabolic insights in disease-relevant states, building the largest and most physiologically relevant datasets in human neurodegeneration.​

Bexorg's platform offers pharmaceutical companies the ability to test almost any therapeutic in metabolically active whole human brains—providing a significant advantage over traditional preclinical models in neuroscience. The technology enables preclinical assessment of drug penetration into the brain, dose-response relationships, toxicity monitoring, and biomarker measurement.​

The company has expanded its proprietary dataset to include hundreds of whole-brain experiments spanning Alzheimer's disease, Parkinson's disease, and other neurodegenerative conditions. This has established one of the world's largest repositories of human CNS data. The company now operates five fully operational BrainEx machines in its downtown New Haven lab, with a sixth in development. With additional machines coming online, Bexorg expects to scale up to 1,000 experiments annually by next year.​

XO Digital: Closing the AI Loop

Beyond the wet-lab platform, Bexorg is developing XO Digital, an artificial intelligence engine that learns from each experiment to create a closed-loop system of in-silico prediction and wet-lab validation. The data generated from BrainEx directly feeds AI models to generate high-fidelity targets, novel biomarkers, and go/no-go decisions with unprecedented accuracy and reliability.​

The company is now training foundation models on petabyte-scale human brain molecular data, creating the first AI system grounded in experimentally measured human neurobiology. This fully integrated platform enables machine learning and AI-centered CNS drug discovery and development at a scale previously impossible.​

By combining comprehensive human datasets with advanced AI models, Bexorg is creating one of the most clinically predictive systems in neuroscience. The platform can validate new targets and biomarkers, understand the pharmacokinetic and pharmacodynamic profiles of drugs, and assess potential safety and effectiveness before clinical trials begin.​

Leadership & Scientific Foundation

Bexorg is led by Dr. Zvonimir Vrselja, M.D., Ph.D., who serves as CEO and Co-founder. Vrselja, a physician-scientist, made the transition from Yale researcher to biotech CEO, bringing both medical expertise and entrepreneurial vision to the company. His co-founder, Dr. Nenad Sestan, remains deeply involved in the company's scientific direction.​

The company's foundational research was published in Nature in 2019, demonstrating that cellular and metabolic activity could be restored to postmortem brains hours after death—a breakthrough that stunned the scientific community. The technology first emerged under a National Institutes of Health project called the Brain Initiative, which aimed to map the human brain to understand more about its architecture.​

Bexorg has assembled a multidisciplinary team spanning engineers, data scientists, bioinformaticians, machine learning experts, physicians, and drug discovery specialists. The company's drug discovery efforts are led by Paul Wes, a former Pfizer executive who drives both industry partnerships and Bexorg's own therapeutic programs. The company has grown from five to 30 employees in just three years.​

Funding & Strategic Growth

Bexorg announced the completion of a $23 million Series A financing led by Engine Ventures, with participation from new investors Connecticut Innovations and E1 Ventures, and existing investors Amplify Partners and Starbloom Capital. The company has raised a total of $42.5 million to date.​

In connection with the financing, Ann DeWitt, Ph.D., General Partner at Engine Ventures, and David Beyer, Partner at Amplify Partners, joined the company's board of directors. Dr. DeWitt emphasized the breakthrough nature of the technology: "Bexorg's whole-human brain platform is redefining CNS drug discovery and enabling asset evaluation at rapid speed. We believe this approach will shorten discovery and development timelines, improve clinical trial success rates, and ultimately bring transformational CNS therapies to patients".​

Proceeds from the financing will be used to expand and optimize the company's AI engine trained on clinically predictive whole-human brain data and advance CNS discovery and development programs through partners and in-licensed assets.​

Strategic Partnerships & Revenue Model

Bexorg's business model centers on research partnerships with pharmaceutical companies, providing a faster, more precise path to new CNS therapies. In mid-2025, the company announced a multi-program research collaboration with Biohaven Ltd. (NYSE: BHVN) to identify, de-risk, and advance next-generation therapies for CNS disorders.​

Biohaven CEO Vlad Coric has been following Bexorg's work for years and called the company "a pioneer" that will "go down in history". Biohaven has already tested two drug candidates on BrainEx—one aimed at boosting brain metabolism and function with age, and another targeting Parkinson's disease. The results gave Biohaven confidence to advance both drugs in development, showing the compounds could reach the right concentrations and act on correct targets in the brain. Coric described the technique as a "fast-forward" that could shave years off the research timeline.​

In 2024, Bexorg also partnered with the University of Oxford and the UK Medical Research Council as part of a collaborative effort to advance translational gene therapy development for neurological diseases. Beyond partnerships, Bexorg develops data products and internal therapeutic programs.​

Market Opportunity & Impact

Today, over 70 million people face devastating neurological diseases such as dementia, Alzheimer's, Parkinson's, and multiple sclerosis. The massive unmet need, combined with the extraordinarily high failure rate of traditional approaches, creates a substantial market opportunity for Bexorg's technology.​

By providing a more accurate, human-relevant model than traditional cellular and animal methods, Bexorg is addressing the root cause of CNS drug failures—the inability to translate preclinical findings to human outcomes. The platform's ability to generate high-fidelity data directly from human brain tissue before clinical trials represents a paradigm shift in neuroscience research.​

The company's approach enables pharmaceutical partners to make go/no-go decisions earlier in development with greater confidence, potentially saving years of time and millions of dollars in failed clinical trials. This value proposition positions Bexorg as critical infrastructure for the entire CNS drug discovery ecosystem.​

Looking Ahead

Bexorg represents a new category of AI-enabled, whole human-brain infrastructure for drug discovery that identifies novel targets and biomarkers, unlocking new biology and opportunities in CNS drug development. The company is building foundational infrastructure for drug discovery, fundamentally transforming how the pharmaceutical industry approaches brain diseases.​

As Vrselja explained, "Through our platform, using donated whole human brains, we restore specific molecular activities essential for drug discovery, while the higher-level brain functions are not restored—honoring each gift by generating a depth of knowledge that could not be gained otherwise". The company operates under rigorous legal framework and bioethics standards, guided by an independent board of experts.​

This ethical framework, combined with breakthrough technology and substantial funding, positions Bexorg to accelerate the development of therapies for neurological diseases that have proven extraordinarily difficult to treat. With its fully integrated platform combining wet-lab experimentation and AI-driven analysis, Bexorg is setting a new standard for CNS translational and discovery research—one that could finally unlock treatments for some of humanity's most devastating diseases.