Our Science

With a diverse patient population and multiple factors driving disease, we know a personalized approach is essential. This is how we identify those most likely to respond favorably to our treatments, as well as translatable disease-relevant biomarkers to efficiently measure our impact early in clinical development.

  • Genetically Defined Diseases

    Our founders established Neuron23 to take advantage of the expanding field of human genetics and develop targeted therapeutics for diseases that have clearly defined genetic contributions.

    We take a machine learning approach to drive a deeper understanding of the contribution of the genes of interest to the underlying biology, and then identify the patients most likely to respond to treatment.

    Our team of drug hunters identify compounds to modulate the pathways of interest and, in parallel, our data scientists bring together large data sets to define the target population.

    A clear example of this is our lead program targeting patients with Parkinson’s disease who carry a LRRK2 mutation, as well as subset of the sporadic PD population, with wildtype LRRK2. We have developed best-in-class small molecule inhibitors of LRRK2 that are now in clinical development.

  • Significant Addressable Population

    Many proteins have genetic associations with diseases in the brain and in the periphery. Therefore we capitalize on our ability to develop brain penetrant and non-brain penetrant molecules to target genetically defined diseases of the central nervous system and those with a systemic contribution.

    Parkinson’s disease is the second most common neurodegenerative disease. The Parkinson’s Foundation estimates that nearly 90,000 people are diagnosed with Parkinson’s disease every year in the U.S., and that 1.2 million people in the U.S. will be living with Parkinson’s by 2030. Neuron23 is developing LRRK2 inhibitors and precision medicine diagnostic tools to enable the ability to potentially impact the progression of disease for greater than 30% of Parkinson’s disease patients, one of

    the largest unmet medical needs that exists today. Given the significant global scale of Parkinson’s disease (< 9 million patients worldwide) our ability to identify and treat 30% of Parkinson’s patients creates a sizeable addressable patient population.

    Likewise, TYK2 is implicated in numerous peripheral autoimmune and CNS disorders with significant unmet medical need. Loss of function mutations in TYK2 are protective for CNS disorders such as multiple sclerosis and peripheral autoimmune disorders such as psoriasis, AS and UC. Neuron23 is developing both CNS penetrant and peripherally restricted TYK2 inhibitors to address both neurological autoimmune and peripheral autoimmune disorders.

  • Patient Stratification

    Our scientific co-founders are leaders in the fields of human genetics and machine learning. By bringing these two disciplines together we are able to identify the patients that have the greatest likelihood of response to our therapeutics.

    By combining public and private data sets, we have developed unique algorithms to stratify patients with the same diagnosis into those likely-to-respond vs not likely-to-respond. We believe this will improve the success rate of our clinical trials and better the lives of patients.

    We have leveraged our core expertise in data sciences to develop the first ever companion diagnostic (CDx) for Parkinson’s disease, in partnership with Qiagen. Through this partnership we will utilize our proprietary CDx in Phase 2 clinical trials in to identify patients with the greatest likelihood of response to our LRRK2 inhibitor, thus bringing the right drug to the right patient. Neuron23 will utilize this same approach for our other programs to identify the indications where our therapies can provide the greatest benefit, and the patients that we will be able to help the most.