Moderna, Vertex collaborate on gene editing therapies for cystic fibrosis

By The Science Advisory Board staff writers

September 17, 2020 -- Moderna and Vertex Pharmaceuticals have entered a three-year research collaboration for the discovery and development of lipid nanoparticles and messenger RNAs (mRNAs) for the delivery of gene editing therapies for the treatment of cystic fibrosis.

Initially, the companies will focus on optimization of lipid nanoparticles and mRNAs that can deliver gene therapies to the lungs, enabling functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to be produced. This is the second collaboration between the companies with the first involving the delivery of mRNA coding for cystic fibrosis protein in the lungs, which is still under development.

Under the agreement, Moderna will conduct research to discover and optimize novel lipid nanoparticles for delivery of gene editing therapies to lung cells for the treatment of cystic fibrosis. Moderna will receive $75 million upfront and will be eligible to receive up to $380 million in development, regulatory, and commercial milestones, plus tiered royalties from any products that result from the collaboration.

Moderna will be responsible for the discovery and manufacturing of lipid nanoparticles and mRNA constructs encoding gene editing endonucleases. Vertex will be responsible for providing other components of the gene editing therapies to be formulated into lipid nanoparticles, as well as subsequent preclinical and clinical development and potential commercialization efforts.

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