During the recent conference of the European Association of Blood and Marrow Transplantation (EBMT), Ionis Pharmaceuticals and Metagenomi announced a collaboration for an ambitious genetic targeting pact. The aim of the agreement is to develop new therapies for patients with blood cancers, solid tumors, and inherited diseases.
Hemophilia A
Earlier this year, Ionis Pharma and Metagenomi Collaboration , Inc. entered into a multi-target research agreement. Under this agreement, Metagenomi will work with Ionis to research and develop four gene-editing therapies.
Metagenomi has been one of the most active companies in the industry when it comes to developing CRISPR-based gene editing technologies. It has developed a series of cell therapy programs for solid cancers, blood cancers, and cystic fibrosis. It has also been a leader in RNA-targeted therapeutics. This collaboration allows Metagenomi to leverage Ionis’ medicinal chemistry, RNA technologies, and alternative routes of delivery. It also gives Ionis an opportunity to use Metagenomi’s expertise in the development of drugs for liver disease.
Several studies have been conducted to evaluate several vector delivery systems for gene therapy. Adeno-associated virus (AAV)-mediated approaches have potential in safety, delivery efficiency, and target specificity. Metagenomi will utilize its cloud computing platform to screen billions of base pairs for new nucleases. It will then use Ionis’ RNA technologies to identify optimal gene editing targets.
Metagenomi’s technology includes an artificial intelligence (AI) cloud computing platform that screens through billions of base pairs to find new nucleases. In addition, Metagenomi has developed a variety of next-generation gene editing tools that can be used to target genes inside the body.
The company has also signed collaborations with Affini-T Therapeutics and Moderna Biosciences. It has also announced plans to expand its Southern California site. Metagenomi expects to have at least 170 employees by the end of the year. It has more than ten drugs in development for metabolic, cardio-renal, neurologic, and infectious diseases.
Ionis and Metagenomi will also work together on novel targets in other tissues. They aren’t disclosing what diseases they are targeting. However, both companies believe that expanding their gene editing technologies will allow them to address a wider range of diseases. This could lead to more patients being treated with their medicines.
This multi-target research deal has the potential to generate nearly $3 billion in biobucks. It’s an ambitious deal for both companies, but it demonstrates the industry’s continued wager on gene editing technologies.
Cystic fibrosis
RNA targeted therapeutics company Ionis Pharmaceuticals has entered a collaboration with Metagenomi, a gene editing technology company, on an ambitious research program. The companies will research up to four genetic targets together. Metagenomi will initially develop medicines for two of the four targets, while Ionis will co-develop the other two. The two companies will begin their work with targets in the liver.
Metagenomi has a deep history in gene editing. Metagenomi was founded by scientists at the University of California, Berkeley. Its technology includes next-generation genome-editing tools and a significant library of enzymes. It is addressing a number of diseases, including cystic fibrosis, hemophilia A, and solid cancers. Its programs also include cell therapy programs for blood cancers. Its artificial intelligence (AI) cloud computing platform screens billions of base pairs to identify new nucleases.
Metagenomi’s partnership with Ionis will provide the company with an opportunity to expand its gene-editing pipeline. Metagenomi will work with Ionis to expand the scope of the collaboration into a second wave of four targets. While the two companies are not ready to disclose the exact targets they are working on, they will focus on de novo targets, which are genetically undefined, and validated liver targets.
Solid and Blood Cancers
RNA-targeted therapeutics company Ionis Pharmaceuticals has entered into a research collaboration with Metagenomi, a next-generation gene editing company. Under the deal, the companies will jointly investigate up to four genetic targets. They will pay Metagenomi an initial $80 million upfront payment, with additional royalties. Eventually, the deal could be worth as much as $3 billion.
The companies plan to focus their work on validated liver targets. However, they are also considering additional targets in extra-hepatic tissues. According to Ionis, the industry is betting heavily on gene editing, and the partnership is part of a larger effort to transform chronic treatments.
Metagenomi will leverage Ionis’ medicinal chemistry and RNA technologies to identify the best targets for gene editing. The company will also use its artificial intelligence-based cloud computing platform to screen billions of base pairs to identify novel nucleases. It also has a large library of enzymes that could be used to target genes.
Ionis and Metagenomi will initially focus on four genetic targets, and the two companies will have the option to add four more targets upon predetermined development milestones. Ionis plans to expand its Southern California site in the near future.
Metagenomi and Ionis were not ready to provide details about the targets. However, Metagenomi CEO Brian Thomas believes there is tremendous opportunity in the deal. He said Ionis has a huge pipeline of RNA-targeted products, and the deal could provide Ionis with a new source of RNA-targeted therapeutics.
Off-Target edits
Earlier this month, Ionis Pharmaceuticals entered into a multi-target research partnership with Metagenomi. The companies will jointly develop and explore four genetic targets. Metagenomi will receive an upfront payment of $80 million and future milestone payments. It will also have the right to add four more targets to the agreement at pre-determined development milestones.
Metagenomi’s partnership with Ionis demonstrates the company’s focus on developing a wider range of gene editing tools and expanding its pipeline. The deal allows Ionis to expand its product portfolio, bringing its antisense technology and antisense therapeutics into a new class of therapies. The collaboration also allows Metagenomi to take advantage of Ionis’ medicinal chemistry and targeted delivery expertise.
The companies will work together to develop four gene-editing therapies. Initially, the deal will focus on liver-based targets. However, Ionis may also consider delivery to extra-hepatic tissues. This option could be advantageous to Metagenomi, which has a strong track record of delivery to extra-hepatic tissues.
Metagenomi’s CEO Brian Thomas is excited about the potential for this collaboration. He believes that expanding the company’s gene editing tools can address more diseases. In addition, he believes that expanding the company’s genetic targeting capabilities will be key to treating a broader range of diseases.
Ionis is a biopharmaceutical company that has developed RNA-targeted therapeutics. Its RNA technologies can be used to identify the best gene editing targets. The company will also focus on developing a range of de novo targets.
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