Cell and gene therapy company ElevateBio pulled off the year’s largest financing, with a $401 million series D round from an investor syndicate. At the same time, its wholly owned gene-editing technology company Life Edit Therapeutics forged a deal with Danish biopharma Novo Nordisk of Bagsvaerd to discover and develop base-editing therapies for rare and cardiometabolic diseases. The agreement could be worth up to $1.9 billion for collaboration on seven development programs.
Life Edit has built a diverse library of RNA-guided nucleases and base editors. The nuclease collection features a range of protospacer-adjacent motifs, which are short sequences that define the segments in the genome where the nuclease can bind, enabling base editing at more sites than those at which one nuclease would bind and providing broad genome access for editing. The platform technology allows ex vivo engineering of cell therapies and regenerative medicines, and in vivo delivery of gene therapies. Novo is shoring up its genetic medicines with the Life Edit deal, adding to its $3.3 billion acquisition of Dicerna Pharmaceuticals in 2021. In February 2023, Moderna also forged a partnership using Life Edit’s tools based on CRISPR technologies to develop in vivo gene editing therapies. As a base-editing company, Life Edit is rival to David Liu’s Beam Therapeutics, though the former’s RNA-guided nucleases are evolutionarily distant from CRISPR–Cas9. At the annual Huntington’s Disease Therapeutics Conference in April in Croatia, Life Edit presented data showing a 40% reduction in mutant huntingtin protein in a mouse disease model using its small RNA-guided nucleases delivered in a single AAV vector.
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