Moderna and Life Edit Therapeutics Enter Gene Editing Collaboration

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In the Lab eNewsletter, Pharmaceutical Technology's In the Lab eNewsletter, March 2023, Volume 18, Issue 3

Moderna will leverage Life Edit’s suite of gene editing technologies to advance development of in vivo gene editing therapies.

Moderna and Life Edit Therapeutics, an ElevateBio company specializing in next-generation gene editing technologies and therapeutics, announced a strategic research and development collaboration on Feb. 22, 2023. The partnership, which will combine Life Edit’s suite of gene editing technologies with Moderna’s messenger RNA (mRNA) platform, will seek to advance in vivo gene editing therapies against a set of various therapeutic targets.

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. According to a company press release, 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, as well as tiered royalties on global net sales of products from the collaboration. The amount of financial compensation was not disclosed in the release.

Life Edit’s gene editing platform offers a library of base editors and RNA-guided nucleases (RGNs). According to the release, the RGNs are smaller in size when compared to conventional nucleases, which the company hopes can enable greater versatility for delivery. Additionally, the company’s nuclease collection features a range of protospacer adjacent motifs, short sequences that determine the DNA segments in the genome to which a nuclease can bind. The diversity of this range is intended to enable base editing at more sites than a single nuclease could achieve. This collaboration will apply Life Edit's RGNs and base editors together with Moderna's mRNA platform with the goal to develop curative therapies for challenging genetic 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, Life Edit Therapeutics, and president, R&D, ElevateBio, in the release. "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."

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"At Moderna Genomics, we are constantly working to accelerate new therapeutic targets that may one day lead to the next generation of transformative mRNA medicines for patients," said Eric Huang, general manager and chief scientific officer, Moderna Genomics, in the release. "Through our collaboration with Life Edit, we hope to harness the power of gene editing technologies as part of our broader research and development engine, helping to advance our mission and deliver on the promise of mRNA."

Source: Moderna