Tag Archives: gene

Modifier Gene May Explain Why Some with Cystic Fibrosis are Less Prone to Infection

Cystic fibrosis is caused by an inherited mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Due to this mutation, the CFTR protein doesn’t embed in cell membranes to form a channel for chlorine ions the way it should. As a result, mucus-producing cells secrete a thicker-than-normal mucus that can create blockages in the lungs and… Read More »

FDA says some data testing Novartis’ $2 million gene therapy was manipulated

NEW YORK (Reuters) – The U.S. Food and Drug Administration said on Tuesday that some data from early testing of Novartis’ more than $ 2 million gene therapy Zolgensma was manipulated, but the agency believes the treatment should remain on the market. FILE PHOTO: The headquarters of the U.S. Food and Drug Administration (FDA) is… Read More »

Novartis building out cell and gene therapy platform that'll lead the pack, CEO promises

SAN FRANCISCO—Novartis’ first-ever CAR-T cell therapy may have been hamstrung by early manufacturing problems, but the company still figures it can build a cell and gene therapy platform strong enough to deliver success not only with Kymriah but also a full “game board” of treatments for many more diseases, its CEO said on Monday. During his first… Read More »