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Biotech Startup Raises $113 Million for Precision Medicine Against Neurodegenerative Diseases 🧬



South San Francisco-based Neuron 23 announced December 17 a $113 million combined Series A and Series B and short-term plans to take on Denali and Biogen in Parkinson's disease and pursue the neural-side of a target that Bristol Myers Squibb has recently pursued to great success elsewhere in the body.


Established through a partnership with Germany’s Origenis, the funds, which includes $30 million in series A funding from Westlake Village BioPartners and Kleiner Perkins and an $80 million series B headed by Redmile Group, will lead two to three programs into the clinic in the next two years.


“We wanted to make sure that when we came out, we were really in the race,” Neuron23 CEO Nancy Stagliano, Ph.D., said. “We thought it was the right time given the focus and resurgence of interests in the CNS industry.”


Since October 2018, scientists at Neuron23 have worked silently on finding their position in the world of precision medicine. The position uses data science and artificial intelligence to target neuroinflammatory and neurodegenerative diseases in patients with certain genetic mutations.


Neuron23’s lead program is aimed at Leucine-rich repeat kinase 2 (LRRK2), a gene that codes for a kinase enzyme of the same name. Mutations in this gene are solidly linked with 3% of patients with Parkinson's disease. What's exciting, Stagliano said, is that detecting a small-molecule inhibitor for LRRK2, which has an obvious genetic connection to the disease, can give a basis for biological knowledge to assist in treating the other 97% of Parkinson's patient.

The second program targets brain-penetrant inhibitors for tyrosine kinase 2 (TYK2), a kinase that causes multiple sclerosis.


However, it is worth noting that getting small-molecule inhibitors into the brain can be difficult. This is largely because the brain has evolved to keep toxins and other pathogens out of it through its blood-brain barrier. Small molecules can get through the bloodstream but can be quickly pumped out from the brain based on their structure or other features.


"One of the strengths of our partnership with Origenis, the additional chemistry partner we founded the company with, is getting these brain-penetrant molecules against these kinds of target kinases," Stagliano said. "LRRK2 is extremely exciting and an area of hot pursuit in our industry because it systematically has been linked to Crohn's disease. So we can look at that second group of LRRK2 molecules that don't get into the brain, and we can say, what can we use those for?”



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