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Pharma Company Gains Precise Gene Edits in $2B Biotech Deal

Editing DNA

(LaCasadeGoethe, Pixabay)

24 May 2023. A global drug maker is acquiring access to a biotechnology company’s precise genome editing techniques to develop new therapies for rare and cardiometabolic disorders. The agreement with Novo Nordisk A/S in Bagsværd, Denmark could bring Life Edit Therapeutics Inc. in Durham, North Carolina and its parent company ElevateBio more than $2 billion if all terms of the deal are fulfilled.

Life Edit Therapeutics discovers new treatments for disease with the genome editing process similar to Crispr, short for clustered, regularly interspaced short palindromic repeats, adapted from a bacterial process for protecting against attacks by viruses. Crispr uses RNA to find the exact location in a genome to edit malfunctioning genes responsible for disease, with enzymes that perform the edits. Life Edit says it extends the Crispr approach with new types of nucleases or enzymes that perform a wider range of genome editing functions. The company says its genome editing enzymes are derived from natural microbial sources provided by its partner company AgBiome Inc.

Among Life Edit’s extended genome-altering techniques is base editing for fixing more granular errors in the genome. Base editing seeks to change errors called point mutations where one base pair in a single DNA strand is replaced by another base pair. The human genome is made up of some 3 billion base pairs — adenine (A)/thymine (T), and cytosine (C)/guanine (G) — and each pair joining with sugar and phosphate molecules to form a nucleotide. The sequence of those nucleotides in strands of DNA make up a person’s genome.

Limit off-target edits and adverse effects

Life Edit says it achieves base editing precision with its highly accurate enzymes designed to cut only a single DNA strand at the specific base-pair location. This level of precision, says the company, is needed to address mutations responsible for rare diseases and limit off-target edits and adverse effects.

Novo Nordisk is a global drug maker and developer of branded drugs for obesity, diabetes, heart disease, and genetic disorders such as hemophilia. The agreement with Life Edit Therapeutics calls for the two companies to collaborate on base editing applied to seven disease targets, including rare genetic diseases and cardiometabolic disorders, where metabolic conditions such as insulin resistance are combined with cardiovascular diseases like hypertension. Under the deal, Novo Nordisk is responsible for all research and development expenses and Life Edit receives an undisclosed up-front payment.

The agreement makes Life Edit eligible for development, regulatory, and commercial milestone payments of up to $335 million for each of the first two therapy programs, and up to $225 million for each of the following five treatments. Life Edit is also eligible for royalties on future sales of therapies. In addition, Novo Nordisk is taking part in the latest venture funding round of ElevateBio, the parent company of Life Edit Therapeutics, a gene and cell therapy holding company in Waltham, Mass. Science & Enterprise reported on ElevateBio’s previous funding round in Mar. 2021.

“The advancements we’ve made,” says Life Edit CEO Mitchell Finer in a statement, “to our next-generation gene editing platform and base editing capabilities are opening the next frontier of treating disease through DNA editing.” Marcus Schindler, executive vice-president and chief scientist at Novo Nordisk adds that his company is “continuously building and leveraging technology platforms that open up new opportunities across our therapeutic areas to deliver potentially curative treatment options to people living with serious chronic diseases.”

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