Case study
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0
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Case study: Discovering Best-in-Class LRRK2 Therapies for Parkinson’s Disease

LRRK2 (G2019S) Inhibitors for Parkinson’s disease. Promising target for Parkinson’s disease (>$5B market). Still no FDA-approved drug targeting LRRK2.

Target

LRRK2 (G2019S)

Chemical space

Enamine REAL 39B

Models we used

B1

Hit rate (<10 μM)

4.6-15.6%

Best hit IC50

0.19 μM

Challenge

Following Alzheimer’s disease, Parkinson’s disease is the second most common neurodegenerative disorder in the US. LRRK2 is one of the most promising drug targets for Parkinson’s disease. There are currently some drug candidates in clinical trials, but no FDA-approved drug. The goal: to pioneer the first approved therapy in this large and critical market.

Solution & Impact

BIOPTIC B1 model quickly identified a chemically novel 0.86 uM LRRK2 inhibitor from a vast chemical space of 39B molecules. In the second iteration we improved potency 4.4 times achieving 0.19uM. This discovery brings us closer to delivering a best-in-class solution for an underserved patient population.

Related

Publications

BIOPTIC B1 Identifies Novel Miro1 ligands for Friedreich's ataxia — Stanford-led Cell Chemical Biology Study

Publications

BIOPTIC Agent Hunt Globally — Wide Search AI Agents for Drug Asset Scouting in Investing, Business Development, and Competitive Intelligence (arXiv, 2026)

Publications

BIOPTIC B1 Ultra-High-Throughput Virtual Screening System Discovers LRRK2 Ligands in Vast Chemical Space

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