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Ionis Pharmaceutical and Metagenomi have entered a collaboration to pursue investigational drugs for up to four validated and novel genetic targets. Ionis is an antisense/RNA-targeting pioneer while Metagnomi specializes in gene editing.

Ionis has the right to add four more targets upon achievement of pre-determined development milestones. Under the terms of the agreement, Ionis will pay $80 million upfront to Metagenomi plus the potential for future milestone payments and royalties.

“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 P. Monia, PhD, Ionis chief executive officer.

“Gene editing has the potential to transform chronic therapies into potentially curative treatments for patients who currently have limited options,” said Brian C. Thomas, PhD, chief executive officer and founder of Metagenomi.

Ionis has three approved drugs and more than ten in mid- or late-stage development. The company is addressing therapeutic areas including cardio-renal, metabolic, neurologic, infectious disease, rare, ophthalmology, pulmonary and allergy and hematology. Their partners include Novartis, AstraZeneca, GSK, Roche, Bayer, and Biogen.

CRISPR (clustered regularly interspaced short palindromic repeats) gene editing is a technology used to modify sequence using a specific RNA-guided nuclease (Cas enzyme). Gene editing enzymes act to correct the genetic code at the precise spot where it has errors.

The technology has already been shown to be promising in conditions including lung cancer and blood disorders. There are more than 70 registered clinical trials for the technology at

“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,” said Monia.

The company’s approved medicines include nusinersen (Spinraza) for Spinal Muscular Atrophy (SMA), inotersen (Tegsedi) for polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTR), and volsnesorsen (Waylivra) for familial chylomicronaemia syndrome (FCS).

The CRISPR system comprises a guide RNA and the nuclease. The guide RNA is complementary to the specific target DNA sequence. The nuclease cleaves the target DNA.

Metagenomi recently announced the opening of a new, in-house Good Manufacturing Practices (GMP) facility, based within its 50,000 square foot campus in Emeryville, California.

The company says its discovery platform has “created the largest library of active nucleases with the ability to edit any codon in the human genome.” This library is the basis for a proprietary gene editing toolbox that “fits a variety of delivery options and is tailored to each therapeutic indication for both in vivo and ex vivo gene editing approaches.”

“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.” said Thomas.

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