Ionis partners with Metagenomi to add gene editing to its broad technology platform

  • Collaboration with leader in gene editing systems is latest move by Ionis to expand and diversify its technology
  • CRISPR-Cas gene editing is a natural extension of Ionis’ innovative approach to delivering potentially transformative therapies
  • Ionis hosts webcast November 14 exist 8 a.m. ET

Carlsbad, California and Emeryville, California, November 14 January 2022 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) and Metagenomi today announced that the two companies have entered into a collaboration that will leverage Ionis’ extensive expertise in RNA-targeted therapeutics and Metagenomi’s Versatile next-generation gene editing systems to pursue combinations of proven and novel genetic targets with the potential to expand treatment options for patients. The two companies will jointly conduct research initially aimed at delivering investigational drugs for up to four genetic targets. Ionis has the right to add four more targets upon achievement of predetermined developmental milestones.

“Ionis was founded more than 30 years ago to discover and develop novel, highly personalized medicines using our powerful RNA-targeting technology platform. The collaboration with Metagenomi supports Ionis’ strategic goals of advancing our technology and expanding our delivery of precision Gene medicine capabilities. Together, we can expand the application of gene editing by leveraging Ionis’ extensive experience in nucleic acid therapy to optimize and expand the impact of gene editing on liver targets and new tissues,” said Ionis CEO Brett P. MoniaDr. “This alliance brings our two companies closer to delivering next-generation therapies with the potential to revolutionize the treatment of diseases.”

“Gene editing has the potential to transform chronic therapies into potentially curative therapies for patients with currently limited options,” said Brian C Thomas, Ph.D., CEO and founder of Metagenomi. “This collaboration is a strategic step to combine Metagenomi’s leading diverse, specific and efficient nuclease gene editing toolbox with Ionis’ pioneering expertise in RNA-targeted therapeutics to catalyze the next wave of in vivo gene editing Applications. We will continue to focus on our strategy of working with the most experienced team in genetic medicine that will allow us to transform patients’ lives.”

Under the terms of the agreement, Ionis will pay $80 million Metagenomi’s upfront costs and potential for future milestone payments and royalties. Wells Fargo Securities acted as financial advisor to Ionis in the transaction.

live streaming

Ionis will be in November 14 exist 8 a.m. ET Discuss this announcement and related events. Interested parties can watch the webcast here. A replay of the webcast will be available at the same address for a limited time.

CRISPR gene editing

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) gene editing is a technique that uses specific RNA-guided nucleases (Cas enzymes) to modify DNA sequences in cells. Gene-editing enzymes act as molecular word processors, correcting the genetic code at the precise locations where they malfunction.

About Ionis Pharmaceuticals, Inc.

For more than 30 years, Ionis has been a leader in RNA-targeted therapeutics, opening new markets and changing the standard of care with its novel antisense technology. Ionis currently has three marketed drugs and a major late-stage pipeline that includes industry-leading cardiovascular and neurology franchises. Our scientific innovation begins and continues with the understanding that patients depend on us, which drives our vision to be a leading, fully integrated biotechnology company.

To learn more about Ionis, visit and follow us on Twitter @ionispharma.

About Metagenomics

Metagenomi is a gene editing company focused on developing potential treatments by leveraging a proprietary toolbox of next-generation gene editing systems to precisely edit DNA that cannot be edited by current technologies. Our metagenomics-powered discovery platform and analytical expertise uncover novel cellular mechanisms derived from otherwise unknown organisms. We have adapted and built these naturally evolved systems into powerful gene editing systems that are ultra-small, extremely efficient, highly specific, and have a reduced risk of immune responses. These systems power our novel drug pipeline and can be leveraged by partners. Our goal is to revolutionize gene editing for the benefit of patients around the world. For more information, please visit

Forward-Looking Statements by Ionis

This press release includes forward-looking statements regarding our collaboration and license agreement with Metagenomi, Inc., the therapeutic and commercial potential of gene targets using gene editing technology, Ionis technology and other Ionis products in development. Any statements describing Ionis’ objectives, expectations, financial or other projections, intentions or beliefs are forward-looking statements and should be considered risk statements. Such statements are subject to certain risks and uncertainties, including risks and uncertainties related to the possible impact of COVID-19 on our business, including without limitation, related to our commercial products and the drugs in our pipeline related risks and uncertainties, especially those inherent in them. The process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and striving to build a business around these medicines. Ionis’ forward-looking statements also involve assumptions that, if they never materialize or prove to be correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements.

Although Ionis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known to Ionis. Therefore, you are cautioned not to rely on these forward-looking statements. These and other risks associated with the Ionis program are described in greater detail in Ionis’ Annual Report on Form 10-K. December 31st March 2021, and our most recent quarterly filings on Form 10-Q with the Securities and Exchange Commission.Copies of these and other documents are available at

In this press release, unless the context otherwise requires, “Ionis,” “Company,” “we,” “our” and “our” refer to Ionis Pharmaceuticals and its subsidiaries.

Ionis Pharmaceuticals® is a trademark of Ionis Pharmaceuticals, Inc.

SOURCEIonis Pharmaceuticals, Inc.

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