Early proof of concept for Krystal’s cystic fibrosis gene therapy: Clinical Report

Beyond headline-grabbing Phase III psoriasis data from Alumis and Arrowhead’s Phase I/II proof of concept for muscle-sparing weight loss, the week also brought early-stage results from Krystal Biotech that could signal progress for cystic fibrosis patients left behind by approved treatments. 

CFTR protein modulators such as Alyftrek, a triple combination of vanzacaftor, tezacaftor and deutivacaftor from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX), have transformed cystic fibrosis (CF) care, delaying lung transplants and improving long-term outcomes. But because these act by improving the function of the mutant CFTR protein, they don’t work for the roughly 10% of CF patients who make no CFTR protein...