Vertex announced that their mRNA therapy IND has been cleared by the FDA

News - 13/12/2022

Vertex Pharmaceuticals have announced that they have approval from the Food and Drug Administration (FDA) to begin clinical trials of their ‘mRNA’ genetic therapy as a treatment for cystic fibrosis. This trial is the result of a collaboration between Vertex and Moderna.

How do mRNA therapies work?

Our DNA is organised into genes, where each gene is a specific instruction for the cell to make a specific protein. For example, the CFTR gene makes the CFTR protein. The actual production of a protein from a gene is a multi-step process, one of the steps involves a protein-making-template called mRNA. mRNA therapies for CF work by adding undamaged protein-making templates for the CFTR protein into cells.

This is encouraging news for people with cystic fibrosis who have gene mutations that do not respond to modulator drugs, such as, Kaftrio. It’s exciting to see innovations that have been developed in the lab now being tested in this early phase clinical trial and we look forward to seeing the results.
David Ramsden, Chief Executive of Cystic Fibrosis Trust

Research into treatments for those not able to benefit from current CFTR modulators is one of the top research priorities recently chosen by the cystic fibrosis community.

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