- Vertex, which markets two gene-therapy based treatments for cystic fibrosis, is teaming up with the well-capitalized Boston-based biotech company Moderna Therapeutics to explore new gene therapy-based approaches to treating the genetic disease, the companies said Wednesday.
- Under the terms of the deal, Moderna will receive $40 million upfront with potential for an additional $275 million in milestones.
- The key to unlocking new treatments, according to Moderna, is through delivering synthetic versions of mRNA proteins. These proteins are intended to enable patients to make their own therapeutic proteins internally.
Vertex has led the way in changing the standard of care for cystic fibrosis (CF) patients with specific genetic mutations. By addressing these mutations, Vertex's existing drugs Kalydeco and Orkambi counteract CF-related lung damage, resulting in fewer infections and less build-up of mucus. These drugs are not curative, however.
The introduction of these drugs within the last several years has been heralded as a major improvement for the roughly 27,000 CF patients worldwide who can benefit from treatment with these agents. With other 70,000 people in the world suffering from CF, however, there is still room for more treatment options.
The companies plan to use Moderna's mRNA platform to enable the lungs to replicate transmembrane conductance regulator (CFTR) proteins. These proteins would be synthetically introduced into the lungs via synthetic mRNA.
Vertex and Moderna are two notable companies. Vertex already has two high priced drugs (list prices range from $259,000 to $311,000) and a long history of collaboration with the CF Foundation.
Moderna, on the other hand, is an extremely well-funded biotech, with roughly $900 million in reserve through venture funding. Recently, Moderna has teamed up with Merck on a cancer vaccine, Alexion on rare liver disease drugs, and AstraZeneca on secondary treatments post myocardial infarction.
“Moderna’s messenger RNA Therapeutics represent a promising new approach that could provide functional CFTR proteins to the lungs of people with CF, which would complement our existing approach of using CFTR modulators to treat CF," said David Altshuler, Vertex's chief scientific officer.