The partners will focus on developing gene editing-based treatments for cystic fibrosis and sickle cell disease, among other diseases.

NEW YORK (GenomeWeb) — Vertex Pharmaceuticals this week released data from two Phase III studies of a combination of its cystic fibrosis drugs lumacaftor and Kalydeco (ivacaftor), showing that the studies have met their primary endpoints of statistically significant improvement in lung function a

Vertex Pharmaceuticals has been awarded

The US Food and Drug Administration last week approved Vertex Pharmaceuticals' Kalydeco (ivacaftor) for cystic fibrosis patients six and older who harbor one of eight mutations in the CFTR gene.

Based on promising results from a late-stage trial, Vertex Pharmaceuticals is hoping to expand the indication of its cystic fibrosis drug Kalydeco (ivacaftor) in other genetically defined patient populations.

European Commission Approves Vertex's Personalized CF Drug Kalydeco

David Rimoin, director of the Cedars-Sinai Medical Genetics Institute, has passed away at age 75 from pancreatic cancer.

David Altshuler has been appointed to the board of directors at Vertex Pharmaceuticals. He is a professor of genetics and medicine at Harvard Medical School, where he has served on the faculty since 2000.

Vertex Pharmaceuticals has announced positive interim results this month from the second part of an ongoing Phase II trial examining a combined regimen of its approved drug Kalydeco and its newer treatment VX-809 in cystic fibrosis patients with the F508del mutation.


360Dx reports that the US Centers for Medicare & Medicaid Services would cover next-generation sequencing-based cancer panel tests.

The Washington Post reports that a meteorologist is being considered as presidential science and technology advisor.

In PNAS this week: precision medicine strategy to screen for disease risk, genome evolution in Haemophilus influenzae, and more.

Researchers have developed a PCR-based assay to gauge whether manatees are present in waters.