Ionis Partners with Metagenomi to Add Gene Editing to its Technology Platform

Rare Daily Staff    

Ionis Pharmaceuticals and Metagenomi have entered a collaboration that will leverage Ionis’ expertise in RNA-targeted therapeutics and Metagenomi’s next-generation gene editing systems to pursue a mix of validated and novel genetic targets that have potential to expand therapeutic options for patients.

“Gene editing has the potential to transform chronic therapies into potentially curative treatments for patients who currently have limited options,” said Brian Thomas, founder and CEO of Metagenomi. “This collaboration is a strategic step to combine Metagenomi’s leading gene editing toolbox of diverse, specific and highly efficient nucleases, with Ionis’ pioneering expertise of RNA-targeted therapeutics, to catalyze the next wave of in vivo gene editing applications. We continue to follow our strategy of partnering with the most experienced teams in the genetic medicines field that will allow us to transform the lives of patients.”

Under the terms of the agreement, Ionis will pay $80 million upfront to Metagenomi plus the potential for future milestone payments and royalties. The companies will jointly conduct research aimed initially at delivering investigational medicines for up to four genetic targets. Ionis has the right to add four more targets upon achievement of pre-determined development milestones.

“This partnership with Metagenomi supports Ionis’ strategic objective to advance our technology and expand our capabilities to deliver precision genetic medicines. Together, we can broaden the application of gene editing by leveraging Ionis’ vast experience in nucleic acid therapeutics to optimize and extend the reach of gene editing for liver targets and to new tissues,” said Brett Monia, CEO at Ionis. “This alliance brings our two companies closer to delivering next-generation therapies with the potential to revolutionize the treatment of diseases.”

Photo: Brian Thomas, founder and CEO of Metagenomi