Vertex Pharmaceuticals
NEWS
With several recent approvals in the space and more on the horizon, BioSpace looks at some of the key decisions and their larger significance both for patients and science.
The U.K. National Institute for Health and Care Excellence on Thursday recommended against funding Vertex Pharmaceuticals’ CRISPR-based sickle cell disease therapy Casgevy unless uncertainties can be cleared up.
Vertex and CRISPR Therapeutics are setting up treatment centers for patients with beta thalassemia and sickle cell disease to compete with bluebird’s established infrastructure.
Two surprise companies, Vertex Pharmaceuticals and Regeneron Pharmaceuticals, made GlobalData’s list with 41.4% and 21.8% market capitalization growth, respectively.
The company’s three-drug combination met all primary and key secondary endpoints in two randomized controlled trials in cystic fibrosis patients, paving the way for an application to regulators.
Positive Phase III results for VX-548 could signal a new chapter in the fight against opioid addiction, but experts say there is still room to do better.
After VX-548 demonstrated significant pain relief in surgical and non-surgical settings, Vertex Pharmaceuticals is preparing to file a New Drug Application for the non-opioid candidate by mid-2024.
Vertex Pharmaceuticals and CRISPR Therapeutics have secured another FDA approval for their Casgevy Cas9 gene-edited cell therapy, this time in treating transfusion-dependent beta thalassemia.
Vertex still has an ongoing diabetes partnership with CRISPR, which gives it a non-exclusive license to the latter’s platform to develop a potential cure for type 1 diabetes.
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