Vertex

A CRISPR Go

US companies have begun recruiting for a CRISPR-based gene-editing trial to treat β-thalassemia, Stat News reports.

Vertex Pharmaceuticals has invested £10.5 million in the British analytics company as the companies launch a multiyear R&D partnership seeking novel drug targets.

This Week in Science

In Science this week: pair of genomics studies on ancient animals, and more.

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

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NPR reports that with medical data being big business, some companies want to get patients involved.

The Asbury Park Press reports on the startup Genomic Prediction's test to determine an embryo's risk of disease.

In PNAS this week: optical mapping allows glimpse of structural variants, disease-linked GATA2 mutations boosts its protein activity, and more.

Senator Elizabeth Warren (D-Mass.) has released the results of a genetic ancestry analysis, the Boston Globe reports.