RARE Daily

Novo Nordisk and Life Edit Enter Gene Editing Collaboration in Rare and Cardiometabolic Diseases

May 24, 2023

Rare Daily Staff

Novo Nordisk and Life Edit Therapeutics, an ElevateBio subsidiary, have entered into a research and development collaboration to discover and develop gene editing therapies against a select set of therapeutic targets.

Novo Nordisk will leverage Life Edit’s suite of gene editing technologies to precisely edit the genome with the aim of developing therapies for rare genetic disorders as well as more prevalent cardiometabolic diseases.

Base editing is a method of gene editing that converts one nucleotide base (a structural component of DNA) into another without cutting both strands of DNA. This is achieved by coupling an enzyme known as a nuclease, modified to cut only one DNA strand, to another enzyme known as a deaminase that edits the target nucleotide base. This precision is intended to reduce the risk of off-target effects and makes base editing a potentially effective approach for correcting genetic mutations associated with diseases.

The collaboration between the two companies allows for the development of up to seven programs. Under the agreement, Life Edit will receive an upfront cash payment and is eligible to receive potential development, regulatory, and commercial milestones of $335 million for each of the first two development programs under the collaboration and up to $250 million for each of the following five development programs under the collaboration. Novo Nordisk will be responsible for all research and development costs and Life Edit is also eligible to receive tiered royalties on future net sales of therapeutic products. In addition, Life Edit has an option to a global profit share on one program.

Novo Nordisk is also making an equity investment in ElevateBio, Life Edit’s parent company, as part of ElevateBio’s $410 million series D financing.

“The advancements we’ve made to our next-generation gene editing platform and base editing capabilities are opening the next frontier of treating disease through DNA editing,” said Mitchell Finer, CEO, Life Edit Therapeutics and president, R&D, ElevateBio. “Combining our Life Edit platform and the ability to make any edit anywhere with Novo Nordisk’s deep disease biology and engineering expertise will bring us closer to delivering potentially curative therapies for some of the world’s most challenging genetic diseases.”

Life Edit is part of ElevateBio’s integrated ecosystem of R&D platform technologies and end-to-end process development and cGMP manufacturing capabilities built to power the creation of life-transforming cell and gene therapies.

The company’s gene editing platform holds one of the world’s largest and most diverse libraries of novel RNA-guided nucleases (RGNs) and base editors that provide flexible editing and unprecedented access to the genome. 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 PAM diversity of Life Edit’s nucleases enables base editing at more sites than any one nuclease could access, enabling the targeting of genetic mutations in tighter windows and providing broad genome access.

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

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