SandboxAQ joins MJFF initiative to target Parkinson’s LRRK2 gene

Company will grant access to its computational, AI tools

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by Andrea Lobo |

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SandboxAQ has signed a $25 million research grant through an initiative of The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to develop new treatments that target the LRRK2 gene, mutations in which are one of the most common genetic causes of Parkinson’s disease.

Under the partnership, which is part of the LRRK2 Investigative Therapeutics Exchange (LITE) initiative, the company will grant access to its computational and artificial intelligence (AI) tools that enable rapid screening of compounds, identifying molecules that could bind intended targets, and designing small molecules.

SandboxAQ will collaborate with the University of Dundee in Scotland to increase the potency of its LRRK2 enzyme inhibitors and identify new compounds that act as allosteric modulators, which are those that regulate enzyme activity by binding to a site other than the active site where a chemical reaction takes place. Key milestones are expected to be delivered over two years.

“By combining MJFF’s network and expertise with SandboxAQ’s AI and computational capabilities, we have a powerful force joining us and creating a unique opportunity to accelerate the development of new therapies for Parkinson’s,” Dario Alessi, PhD, director of the MRC Protein Phosphorylation Unit at the University of Dundee and LITE program leader, said in a press release. “This collaboration represents a significant step forward in our understanding and treatment of this challenging disease.”

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G2019S mutation carriers in LRRK2 gene have higher Parkinson’s risk

Targeting LRRK2

The LRRK2 gene’s enzyme plays an important role in cellular communication and regulating recycling and waste-disposal cellular mechanisms. Mutations in the gene lead to the enzyme being excessively activated, resulting in cellular dysfunction and Parkinson’s development.

Although several treatments targeting LRRK2 are undergoing clinical trials and several other candidates have been identified, no therapies have been formally approved.

The LITE program will connect companies with academic and clinical researchers to develop projects that focus on translating basic science into drug development. Besides establishing best practices for preclinical and clinical studies, LITE is also designed to implement infrastructure to test new LRRK2 biomarkers.

SandboxAQ’s large quantitative models integrate AI tools with physics-based methods that can help accurately predict the behavior of molecules, combine predictions with biologically relevant mechanisms, and generate new molecules with desired properties.

This is the second research partnership the company has established with MJFF. It’s also collaborating with KU Leuven’s Vangheluwe Lab in Belgium to identify small molecules to restore the function of the ATP10B gene, which is linked to a higher risk for developing Parkinson’s disease.