Novo Nordisk is continuing its push into genetic medicines, agreeing to pay NanoVation Therapeutics up to $600 million to collaborate on up to seven programs built on technology for targeting cells outside the liver.
The Danish Big Pharma has shifted the focus of its pipeline in recent years. Having made its name with peptides and proteins, the company has expanded its pipeline to cover modalities including small molecules, RNAi therapies and gene editing. Novo has used many of the novel modalities as part of its concurrent move deeper into rare diseases.
The NanoVation deal reflects the shift in Novo’s focus. The pharma has secured a license to use NanoVation’s long-circulating lipid nanoparticle (LNP) technology in the development of two base-editing therapies in rare genetic diseases. The deal covers up to five more targets in rare and cardiometabolic diseases.
NanoVation has extended the systemic circulation of its LNP to facilitate efficient delivery to cells outside of the liver, including to tissues such as bone marrow, tumors and skin. The biotech published a paper on the technology one year ago, showing how changing the lipid composition of a LNP can slow the rate at which it is cleared to the liver.
Novo is paying an upfront fee of undisclosed size to enter into the collaboration. Factoring in milestones, the deal could be worth up to $600 million plus research funding and tiered royalties on product sales.
The decision to work on the two rare diseases first and then potentially add cardiometabolic targets to the collaboration is in line with Novo’s broader approach to novel modalities. At the company’s capital markets day in March, Martin Lange, M.D., Ph.D., executive vice president, development, at Novo, said the company could “start out testing and learning in the rare disease space” before expanding its use of technologies such as gene editing into larger indications.