Moderna, Life Edit To Develop In Vivo Gene Editing Therapies

Moderna has tapped Life Edit Therapeutics, an ElevateBio company, to discover and develop in vivo mRNA gene editing therapies. 

The multi-target partnership will combine Life Edit’s gene editing technologies, including base editing, with Moderna’s mRNA platform to develop in vivo mRNA gene editing therapies against an undisclosed set of targets. 

“This collaboration between Life Edit and Moderna demonstrates the strength of our respective technologies to advance programmable medicines to more specifically target disease,” said Mitchell Finer, Ph.D., CEO of Life Edit Therapeutics and President of R&D at ElevateBio. 

“Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development. We are excited that partners, such as Moderna, are recognizing the potential of our technology.”

Moderna began the year with its first-ever acquisition of Japanese DNA manufacturer OriCiro Genomics for $85 million. The purchase adds cell-free DNA synthesis and plasmid DNA amplification to Moderna’s repertoire, which could potentially accelerate its R&D engine. 

Related article: Gene Repair Therapy: First Patient Dosed With New Treatment for Sickle Cell Disease 

Base Editors and RNA-guided Nucleases

Life Edit’s gene editing platform offers a library of base editors and RNA-guided nucleases (RGNs). RGNs are smaller than conventional nucleases, which may enable greater versatility for delivery. 

The company’s nuclease collection includes a range of Protospacer Adjacent Motifs (PAMs), short sequences that determine the DNA segments in the genome to which a nuclease can bind. According to Life Edit, the diversity of their PAMs enables base editing at more sites than any nuclease can achieve, which could offer unprecedented access to the genome to target diseases.  

Under the collaboration, Moderna will fund research and preclinical studies by Life Edit. Upon opting in on a target, Moderna will take over further development, production, and commercialization. Life Edit will receive an undisclosed upfront sum and could receive potential milestone payments as well as tiered royalties for each target chosen by Moderna. 

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Joy Lin

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