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Moderna picks up a $75M cash windfall as longtime collaborator Vertex signs up for a gene editing assault on cystic fibrosis

After nailing down the top drug franchise in cystic fibrosis, Vertex $VRTX is turning to the mRNA aficionados at Moderna $MRNA to use gene editing tech for a generation 2.0 attempt to master the disease. This is their 3rd pact together in 4 years as they jointly set their sights on the patients who are toughest to treat.

Researchers at Moderna and Vertex will now work together to develop lipid nanoparticles and mRNA to deliver gene editing tools to cells in the lung to spur production of functional cystic fibrosis transmembrane conductance regulator (CFTR) protein.

Moderna — one of the leaders in the race to develop a vaccine for Covid-19 — gets $75 million up front and up to $380 million in milestones, plus tiered royalties for anything that gets over the finish line at the FDA. And there’s another $25 million coming in from Chiesi, which is partnering with Moderna on an mRNA approach to pulmonary arterial hypertension.

Together, the two deals are being used to chum the waters for Thursday’s R&D day at Moderna, helping send a clear message that the biotech has a broad landscape of drugs it wants to develop — aside from the Covid-19 vaccine work that has attracted close global attention. After the pandemic is over, Moderna intends to make itself into a major player, operating alongside the biggest biotechs in the business.

The Vertex deal marks an addendum to the original collaboration on CF between these two companies to develop mRNA drugs for the disease. They initially signed up for a partnership 4 years ago, then extended the pact in 2019.

David Altshuler, the CSO at Vertex, put it like this today:

Vertex’s CFTR modulator therapies have the potential to treat the vast majority of CF patients and address the underlying cause of their disease. However, approximately 10 percent of patients do not produce any CFTR protein and so are unlikely to benefit from our existing medicines. Over the past 5 years, we have made important progress in our research efforts aimed at the creation of genetic therapies for CF, with the delivery of such therapies remaining the most significant technological and scientific challenge. The combination of Moderna’s unique expertise in the discovery and manufacturing of novel LNP delivery systems and mRNA technologies, combined with Vertex’s scientific, clinical and regulatory capabilities in CF, will accelerate the development of groundbreaking genetic therapies for people with CF and supports our commitment to developing therapies for all people living with CF.