Vertex Pharmaceuticals
NEWS
Eli Lilly has secured the rights to Verve Therapeutics’ gene editing approach, a “one-and-done” method that the companies hope will lower the cardiovascular risk factor lipoprotein(a).
Data show the potential of Editas’ sickle cell disease and transfusion-dependent beta thalassemia gene therapy candidate, but it might not be enough to overtake Vertex and CRISPR Therapeutics.
Drugs that act on the CFTR protein only work in patients who produce the protein in the first place. That leaves 6% of patients hanging.
The regulatory filing puts the company alongside Vertex and CRISPR, which also await FDA approval for their SCD gene therapy.
Currently, there are no gene editing–based treatments on the market, but the technology continues its march toward potential FDA approval, with several products in mid- and late-stage trials.
In a potential leap forward for sickle cell disease, Vertex moved closer to launching its one-time treatment for sickle cell disease and transfusion-dependent beta-thalassemia.
Partners CRISPR and ViaCyte (Vertex), and Genprex are pioneers in the quest to develop a gene therapy for Type 1 diabetes.
Vertex Pharmaceuticals is marching toward the launch of exa-cel with a lot of confidence, per its full-year 2022 presentation.
Amid growing demands for generic versions of Vertex’s Trikafta, the company provided an update on the late-stage clinical development of its next-in-class triple combination for cystic fibrosis.
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