- Already well versed in using messenger RNA to create vaccines, Moderna, through its latest partnership, hopes to use that technology to develop therapies which edit genes “in vivo,” meaning inside a patient rather than in a lab.
- The deal between Moderna and Life Edit Therapeutics, announced Wednesday, will pair the former’s mRNA platform with the latter’s “suite of proprietary gene editing technologies,” which includes DNA base editors and RGNs, or RNA-guided nucleases. While Moderna didn’t disclose therapeutic targets, it did note how gene editing technology “has the potential to treat or cure rare genetic and other diseases.” Financial terms also weren’t disclosed.
- Under their agreement, the companies will collaborate on research and preclinical studies. Moderna will fund those studies, and can take control of a program after a therapeutic target is selected, at which point it would be responsible for further development, manufacturing and commercialization.
While the success of its COVID-19 vaccine made Moderna a household name, it has long had ambitions beyond preventive shots. Today, a significant portion of the company’s long list of research programs is dedicated to treating diseases.
Moderna sees gene editing as a valuable technology for some of those diseases. In 2020, it kicked off a three-year research collaboration with Vertex Pharmaceuticals, aiming to discover ways to deliver gene editing therapies to the lungs of cystic fibrosis patients. Then, in late 2021, Moderna formed an alliance with Metagenomi that gave it access to the startup’s CRISPR-based gene editing tools.
With Metagenomi and, now, Life Edit, Moderna is particularly interested in therapies that work “in vivo.” By directly altering genes in a patient, these therapies could, at least in theory, sidestep many of the biological and logistical challenges that their “ex vivo” counterparts face.
In a statement, Moderna said Life Edit’s technology offers a “large and diverse library” of gene editing tools. Its RGNs, for example, are “smaller in size when compared to conventional nucleases, potentially enabling greater versatility for delivery.”
Life Edit also has a variety of protospacer adjacent motifs, which, Moderna argues, could support base editing at more sites on a gene.
Based in North Carolina, Life Edit was created by AgBiome and then later acquired by ElevateBio, a well-funded biotechnology company focused on the manufacturing of cell and gene therapies.
ElevateBio has a long list of backers, including MPM Capital, F2 Ventures, EcoR1 Capital and The Invus Group, and in 2021 raised more than half a billion dollars through a Series C financing round. Last year, the company locked down a 30-year partnership with the University of Pittsburgh and launched a cell therapy-focused startup with Boston Children’s Hospital.