• Collaboration with a leader in gene editing systems is Ionis’ latest move to expand and diversify its technology
  • CRISPR-Cas gene editing is a natural extension of Ionis’ innovative approach to delivering potentially transformative therapies
  • Ionis to hold webcast Nov. 14 at 8 a.m. Eastern Time

CARLSBAD, CA and EMERYVILLE, CA, USA I November 14, 2022 I Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Metagenomi today announced that the companies have entered a collaboration that will leverage Ionis’ extensive expertise in RNA-targeted therapeutics and Metagenomi’s versatile next-generation gene editing systems to pursue a mix of validated and novel genetic targets that have potential to expand therapeutic options for patients. 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.

“Ionis was founded over 30 years ago to discover and develop novel, highly personalized medicines using our powerful RNA-targeting technology platform. 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 Ionis Chief Executive Officer Brett P. Monia, Ph.D. “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 treatments for patients who currently have limited options,” said Brian C. Thomas, Ph.D., chief executive officer and founder 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. Wells Fargo Securities acted as financial advisor to Ionis on the transaction.


Ionis will conduct a webcast on Nov. 14 at 8 a.m. Eastern Time to discuss this announcement and related activities. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address.

CRISPR Gene Editing

CRISPR (clustered regularly interspaced short palindromic repeats) gene editing is a technology that modifies the sequence of DNA in cells utilizing a specific RNA-guided nuclease (Cas enzyme). Gene editing enzymes act as molecular word processors to correct the genetic code at the precise spot where it is malfunctioning.

About Ionis Pharmaceuticals, Inc.

For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading cardiovascular and neurological franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming a leading, fully integrated biotechnology company.

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

About Metagenomi

Metagenomi is a gene editing company committed to developing potentially curative therapeutics by leveraging a proprietary toolbox of next-generation gene editing systems to accurately edit DNA where current technologies cannot. Our metagenomics-powered discovery platform and analytical expertise reveal novel cellular machinery sourced from otherwise unknown organisms. We adapt and forge these naturally evolved systems into powerful gene editing systems that are ultra-small, extremely efficient, highly specific and have a decreased risk of immune response. These systems fuel our pipeline of novel medicines 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 https://metagenomi.co/.

SOURCE: Metagenomi