Moderna has landed R&D deals with Vertex and Chiesi worth $100 million upfront. Vertex struck the larger of the two pacts, agreeing to pay Moderna $75 million to collaborate on gene editing cystic fibrosis therapies.
Through the Vertex agreement, Moderna will discover and optimize lipid nanoparticles (LNPs) for the treatment of cystic fibrosis. Using the LNPs, the partners aim to get gene editing therapies to lung cells and, in doing so, enable the expression of functional cystic fibrosis transmembrane conductance regulator (CFTR) protein.
In a statement, Vertex Chief Scientific Officer David Altshuler, M.D., Ph.D., called the delivery of genetic therapies for cystic fibrosis the “most significant technological and scientific challenge.” Vertex is looking to Moderna to help it overcome that challenge.
Vertex is paying Moderna $75 million upfront to enter into the collaboration and committing to up to $380 million in milestones. In return, Moderna will discover and produce LNPs and mRNA constructs encoding gene editing endonucleases. Vertex will handle some preclinical and all clinical work.
The deal comes four years after Vertex tasked Moderna with developing mRNA therapies capable of enabling lung cells to make functional CFTR protein. The new project will try to achieve the same goal using LNPs to deliver gene editing therapies. Both programs are part of Vertex’s effort to help the minority of cystic fibrosis patients who do not make any CFTR and, as such, are unlikely to benefit from its existing medicines.
Moderna disclosed a separate deal with Chiesi shortly before sending out the Vertex statement. The agreement will see Chiesi pay $25 million and commit to up to $400 million in milestones to partner with Moderna on the development of mRNA treatments for pulmonary arterial hypertension (PAH).
Johnson & Johnson made a big play for the PAH market in 2017 when it paid $30 billion to buy Actelion. However, Chiesi sees an unmet need for medicines capable of delaying or reversing the disease, leading it to turn to Moderna for support. The collaboration will pair Moderna’s mRNA capabilities with Chiesi’s knowledge of PAH biology.