Moderna and Life Edit Therapeutics look to speed up gene editing therapies

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mRNA

Moderna, Inc., a biotechnology company working on messenger RNA (mRNA) therapeutics and vaccines, and Life Edit Therapeutics Inc., an ElevateBio company focused on gene editing technologies and therapeutics, have announced 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. mRNA may hold promise in the delivery of gene-editing technology, which has the potential 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, chief executive officer, Life Edit Therapeutics and president, R&D, 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.”

Greater versatility

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 access to the genome to target disease.

“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. 

“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.”

Moderna and Life Edit Therapeutics’ collaboration details

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

Under the agreement, Life Edit Therapeutics and Moderna will collaborate on research and preclinical studies, which will be funded by Moderna. 

Upon exercising an option for a target, Moderna will assume responsibility for further development, manufacturing, and commercialization. Life Edit Therapeutics 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.