Moderna Moves Into Gene Editing Space with Metagenomi R&D Collab
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Cambridge, Mass.-based Moderna and Emeryville, Calif.-based Metagenomi inked a strategic research and development deal on new gene editing systems for in vivo (in the body) human therapies. The partnership will leverage Metagenomi’s novel gene editing technology and Moderna’s mRNA platform, in addition to its lipid nanoparticle (LNP) delivery technologies. The focus will be on serious genetic diseases.
No financial details were announced, but Moderna is paying Metagenomi cash up front. Metagenomi will be eligible for target option exercise fees, development, regulatory and commercial milestones, and tiered royalties on net sales of any commercialized products that come out of the collaboration. Moderna is also investing in the company in the form of a convertible note.
Moderna, as everyone knows, focuses on messenger RNA (mRNA) technology. Currently, its sole commercial product is a COVID-19 vaccine, one of only three authorized in the United States.
Metagenomi focuses on developing next-generation gene editing systems. It has a proprietary discovery engine that starts with vast amounts of metagenomic data “to unlock 4 billion years of microbial evolution and identify new types of gene editing systems.”
The company uses advanced artificial intelligence (AI)-based cloud computing to identify and improve natural enzyme systems that may have applications to treating disease. Metagenomi also indicates it has already evaluated hundreds of novel gene editors and validated them in human therapeutic development.
At this time, Metagenomi’s pipeline is entirely in research or lead optimization. Its in vivo targets include primary hyperoxaluria Type 1 (PH1), hemophilia A, four different unspecified liver targets, and cystic fibrosis. Under cell therapies, it has four categories of immuno-oncology programs in solid tumors and blood cancers, including T cell receptors (TCR), CAR-T, Modular CAR and induced Pluripotent Stem Cells (iPSCs).
“Gene editing has the potential to provide a cure for millions of patients living with genetic disease,” said Brian C. Thomas, chief executive officer and co-founder of Metagenomi. “Our partnership with Moderna is designed to accelerate the creation of genetic medicines using Metagenomi’s naturally derived, compact, modular and precise gene editing systems. This partnership will enhance our shared vision to forge transformative therapeutics for patients.”
Although Moderna’s overall pipeline has been overshadowed by the success of its COVID-19 vaccine, on October 26, it announced it had dosed the first participant in its Phase III pivotal registration trial of its mRNA vaccine against cytomegalovirus (CMV).
CMV is a latent virus that stays in the body after infection for the rest of your life. In adults, initial infection is often undetected, and in healthy people, it isn’t a long-term health risk. But for individuals with weakened immune systems or infants born with CMV infection, it has serious implications.
Infants born with congenital CMV infections may have long-term health problems like hearing loss, vision problems, learning disabilities and decreased muscle strength and coordination. Some will become ill at birth with jaundice, rash, liver and spleen enlargement, and seizures. It is the leading infectious cause of congenital disabilities in the U.S.
Moderna’s pipeline also includes vaccines against Epstein-Barr virus (EBV), HIV, human metapneumovirus, Zika virus, seasonal influenza, and therapies for propionic and methylmalonic acidemia, cancer and others.
Eric Huang, general manager and chief scientific officer of Moderna Genomics, said, “Metagenomi has demonstrated the power of its proprietary metagenomics approach that mines the Earth’s natural environment to discover next-generation gene editing tools and has developed discovery capabilities with the potential to address multiple disease. Their discovery platform and expertise will expand Moderna Genomics’ ongoing efforts to develop innovative in vivo gene editing therapies to address a significant unmet medical need.”