A new research project focusing on rare genetic mutations that are linked to cystic fibrosis just received $600,000 in ...

The past decade has been a game-changing one for Vertex Pharmaceuticals (NASDAQ: VRTX). The biotech won approval for its ...

and additional approaches for rare CFTR mutations not yet addressed Core Function #2: Conduct and develop endpoint measures to assess CFTR function, epithelial physiology, preclinical endpoints, and ...

In their 2019 paper, Liu’s team used prime editing to alter the gene mutations causing sickle cell disease and Tay-Sachs disease. Liu explained that they attempted to edit mutated CFTR as well but ...

Cystic fibrosis (CF) is a life-threatening autosomal recessive disease affecting over 160,000 people worldwide. CF is caused by loss-of-function mutations in the CF transmembrane conductance regulator ...

Medically reviewed by Reza Samad, MD Cystic fibrosis (CF) treatment typically involves procedures and medications that help clear the airways. Other treatments may include antibiotics, pancreatic ...

[1] These mutations are called gating mutations. The CFTR protein reaches the cell membrane but the channel does not open properly and chloride transport cannot occur (Figure 1d). Class IV ...

Together these drugs cover the CFTR mutations seen in around half of all CF patients. The next phase of Vertex is focused on the roll-out of its new triple Kaftrio (tezacaftor, ivacaftor and ...

Normally, these fluids are slick and thin. However, mutations in the CFTR gene in people with CF make the secretions thick and sticky, resulting in infection in the airways, clogged ducts and ...

UK MHRA approves Vertex’s Alyftrek, a once-daily next-in-class CFTR modulator to treat cystic fibrosis: London Monday, March 10, 2025, 10:00 Hrs [IST] Vertex Pharmaceuticals, a ...

CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these ...

given its promise to treat all mutations of the disease, including those that result due to the absence of CFTR expression in some patients," said Bhanu P. Jena, Chairman of Porosome Therapeutics ...