Vertex Pharmaceuticals
NEWS
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.
Vertex and Arbor Biotechnologies extend partnership in precision gene editing.
2022 featured pivotal data that could change the landscape in non-alcoholic steatohepatitis, Alzheimer’s disease, sickle cell disease and many more indications.
The FDA lifted its partial clinical hold on bluebird’s experimental sickle cell disease gene therapy for patients younger than 18.
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