23andMe has received a $300 million equity investment from GlaxoSmithKline (GSK) under a collaboration designed to use genetic data to identify new drug targets, and thus accelerate development of new therapies from both companies.

The collaboration is intended to combine 23andMe’s expertise in gathering and analyzing data from the 5 million customers in its research platform—which the company says includes the world’s largest consented, re-contactable database—with GSK’s know-how in R&D and commercialization.

GSK has disclosed one potential new therapy it plans to study with 23andMe—a small-molecule Leucine-Rich Repeat Kinase 2 (LRRK2) inhibitor now in preclinical development for Parkinson’s disease, having been implicated in the autosomal dominant form of the disorder.

The inhibitor of the LRRK2 mutated protein appears in only 1% to 2% of people with Parkinson’s, the second-most common neurodegenerative disease affecting roughly 10 million people worldwide—1 million of them in the U.S. according to the Parkinson’s Foundation. Two hundred-fifty customers of 23andMe reportedly have the disease.

“The idea that there’s 250 patients in the 23andMe database, most of whom have probably consented to research, and for whom there might be an available therapy, but would have taken maybe many, many years to conduct a trial, could be done significantly faster,” Hal Barron, M.D., GSK’s CSO and president, R&D, told reporters during a conference call. “We can really speed up the development time as well, making us very excited about the opportunity of pursuing this.”

Added Anne Wojcicki, CEO and co-founder of 23andMe: “With over 80% of customers consenting to participate in research, it’s really clear that a lot of our customers want to play an active role in discovering and developing treatments and cures for diseases.”

Last year, GSK terminated a Phase I observational study (NCT01424475) designed to assess the phenotypic neurocognitive abnormalities of Parkinson’s patients with the LRRK2 mutation—with the aim of identifying potential PD endpoints related to the LRRK2 mutation for future Phase I or II clinical trials of LRRK2 inhibitors.


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