New Research Partnership Seeks Ways to Modify Patients’ Genes

New Research Partnership Seeks Ways to Modify Patients' Genes

( – Scientists have been looking for cures to some of the worst genetic diseases for years. For instance, the National Institutes of Health (NIH) has a study dedicated to treating Sickle Cell Disease through gene therapy. Pharmaceutical giant Moderna has partnered with another company to develop more possible treatments.

On February 22, Moderna announced it was partnering with ElevateBio’s Life Edit Therapeutics Inc. to develop gene editing technologies. The two companies would use Moderna’s mRNA platform in conjunction with Life Edit’s technology, including its base editing, to “advance potentially life-transformative or curative therapies for some of the most challenging genetic diseases.”

ElevateBio CEO Mitchell Finer said the collaboration between the two companies shows the strength of their individual technology advancements to be able to target diseases in a more nuanced manner.

Messenger RNA (mRNA) is necessary for protein production. Moderna’s technology works by instructing the cells to make proteins that trigger an immune response to target the disease in question. Life Edit, on the other hand, has RNA-guided nucleases (RGNs) and base editors. These RGNs aren’t as large as average nucleases, possibly allowing a “greater versatility for delivery.” The company also has a variety of Protospacer Adjacent Motifs (PAMs). These are short sequences that figure out what part of the genome’s DNA segments the nuclease can bind to.

Moderna and Life Edit will work with one another on preclinical studies and research that the former will fund under their agreement. The pharmaceutical company will be responsible for the manufacturing, future development, and commercialization of anything created. Life Edit would receive a payment and be eligible to receive more regulatory, potential development, and commercial milestone payments.

The companies hope to create revolutionary therapy to help patients across the country and the world.

Eric Huang, Moderna Genomics’ chief scientific officer and general manager, said the partnership with advance the company’s “mission and deliver on the promise of mRNA.”

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