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Ionis Pharmaceuticals, the California company that turned RNA-targeting medicines from an unproven idea into blockbuster drugs, is now expanding into a new class of therapeutics that treat disease by editing DNA. 

On Monday, the company announced a partnership with Metagenomi to kickstart these efforts. Ionis will pay the Bay Area gene-editing startup $80 million upfront as the companies work together to develop up to four gene-editing therapies, with the option to add four more targets. Their initial plan is to focus on the liver, though the firms have yet to disclose precisely what diseases they’ll pursue. 


Executives for Ionis and Metagenomi also declined to elaborate on the total possible future value of the deal, often known as the “biobucks” value, which would include milestone payments. But Ionis CEO Brett Monia was clear about how he’ll evaluate the five-year partnership’s success: multiple drugs either in clinical trials or with proof-of-concept studies in the lab supporting their safety and effectiveness.

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