Can anyone catch Vertex? Inside the cystic fibrosis pipeline

In recent decades, the pharmaceutical industry has developed next-generation treatments for cystic fibrosis (CF) that have a real impact on patients’ lives and extend life expectancy considerably.

There are between 70,000 and 100,000 sufferers of the rare genetic disease worldwide and some 30,000 in the USA alone. CF affects one in 3,000 new-borns and both parents must be carriers of the faulty CF transmembrane (CFTR) gene for the child to develop the condition.

The disease affects the gene which controls the movement of salt and water in and out of cells. It causes the body to produce thick mucus, which affects the lungs and digestive and reproductive systems in particular, and the condition typically worsens over time, leading to eventual fatality.