RARE Daily

Moderna and Elevate’s Life Edit Therapeutics Enter Strategic In Vivo Gene Editing Collaboration

February 22, 2023

Rare Daily Staff

Moderna and ElevateBio’s Life Edit Therapeutics said they have entered into a strategic research and development collaboration to discover and develop in vivo mRNA gene editing therapies.

The partnership will combine Life Edit’s suite of proprietary gene editing technologies, including base editing, with Moderna’s mRNA platform to advance in vivo gene editing therapies against a select set of therapeutic targets. The companies believe mRNA may hold promise in the delivery of gene-editing technology to treat or cure rare genetic and other diseases.

“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, CEO of Life Edit Therapeutics and president of R&D for 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.”

Life Edit’s gene editing platform offers a large and diverse library of base editors and RNA-guided nucleases (RGNs). The RGNs are smaller in size when compared to conventional nucleases, potentially enabling greater versatility for delivery. Life Edit’s nuclease collection features a range of Protospacer Adjacent Motifs (PAMs), short sequences that determine the DNA segments in the genome to which a nuclease can bind. The diversity of Life Edit’s PAM collection enables base editing at more sites than any one nuclease could achieve, offering unprecedented access to the genome to target disease.

This collaboration will apply Life Edit’s diverse collection of novel RNA-guided nucleases (RGNs) and base editors together with Moderna’s mRNA platform with the goal to develop curative therapies for some of the most challenging genetic diseases.

Under the agreement, Life Edit and Moderna will collaborate on research and preclinical studies funded by Moderna. Upon exercising an option for a target, Moderna will assume responsibility for further development, manufacturing, and commercialization. Life Edit will receive an upfront payment and is eligible to receive potential development, regulatory, and commercial milestone payments for each target for which Moderna exercises an option, in addition to tiered royalties on global net sales of products from the collaboration. The companies did not provide additional financial details.

Photo: Mitchell Finer, CEO of Life Edit Therapeutics and president of R&D for ElevateBio

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