• Otávio Santiago

Biotechs Carve out Niches in Cystic Fibrosis Treatment Space


Cystic fibrosis has proved resistant to treatments that provide long-lasting respite from the sticky mucus that affects the lungs and other organs. Emerging biotechs such as Kither Biotech as well as the old guard including Vertex are finding new ways to tackle the inherited disease.


Cystic fibrosis is best known for causing thick mucus in the lungs that can clog the airways and trap germs, rendering it difficult to breathe and facilitating respiratory infections. The inherited condition is caused by mutations in the gene that produces the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which regulates the flow of water and salt components in and out of cells.


Treatment for the more than 75,000 people affected worldwide includes several drugs called CFTR modulators that help the mutated CFTR protein work more effectively.

The undoubted leader in CFTR modulators is US behemoth Vertex Pharmaceuticals. The company markets several drugs that have been hailed as game-changers in the field, such as Orkambi. However, existing drugs haven’t completely solved the excessive buildup of mucus in cystic fibrosis, and can only help patients with certain mutations.


Enter Italian startup Kither Biotech, which raised €18.5M in a Series B financing last week. Kither’s first-in-class inhaled peptide is designed to enhance existing cystic fibrosis treatments by acting on the disease in an entirely new way. Rather than targeting CFTR directly, the drug improves the protein’s function by increasing the levels of a messenger molecule in the cell called cyclic adenosine monophosphate (cAMP).


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