Vertex Signs Letter of Intent (LOI) with the pan-Canadian Pharmaceutical Alliance for ALYFTREK for the Treatment of Cystic Fibrosis

Vertex Pharmaceuticals today announced that it has signed a Letter of Intent (LOI) with the pan-Canadian Pharmaceutical ...

Personalized disease-in-a-dish can improve a pancreatitis patient's therapeutic path

Around 3 million people worldwide struggle with chronic pancreatitis, a condition in which the pancreas becomes inflamed, ...
Phys.org

Nanobody repairs misfolded CFTR inside cells, boosting function in cystic fibrosis

A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers have succeeded in developing a so-called nanobody that penetrates directly ...
Labroots

A Gene Editor to Correct a Common Cystic Fibrosis Mutation

Cystic fibrosis (CF) is a common genetic disorders that has been well studied. Researchers have identified CF-causing mutations in a gene called CFTR, which encodes for an ion channel. The genetic ...
BioWorld

Adenine base editing corrects G542X mutation in CFTR in patient organoid models

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 ...
Los Angeles Times

Genetic medicine can leave people with rare mutations behind. But there’s new hope

Emily Kramer-Golinkoff can’t get enough oxygen with each breath. Advanced cystic fibrosis makes even simple things like walking or showering arduous and exhausting. She has the most common fatal ...