Vertex’s latest treatment for cystic fibrosis is a “triple combination” drug called Trikafta, which like others from Vertex boosts the activity of a protein that’s defective in people with the disease. It has quickly become a go-to treatment, entrenching Vertex’s dominant market position.
Other companies have tried to create competing treatments that can match Vertex’s medicines, but have so far fallen short.
Still, even Trikafta doesn’t work for a minority of patients, leaving an opportunity for other developers. Carbon is one, and has plans to develop an unorthodox gene therapy solution.