Researchers from the Broad Institute, Mass General, Stanford, and elsewhere are investigating why patients with South Asian ...

The company will use the proceeds to test its gene therapy XC001 in two Phase II studies involving patients with cardiovascular disease.

The firm has discussed positive Phase I results on BCB-276 with the FDA and plans to move it directly to a registrational trial by year-end.

Chinese regulators approved the drug for the same subset of advanced, previously treated EGFR-mutated NSCLC patients in 2023.

Vertex aims to further bolster its gene-editing therapy pipeline by using Orna's lipid nanoparticle delivery technologies during the three-year collaboration.