Vertex Pharmaceuticals
NEWS
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.
Vertex is kicking off a Phase I trial assessing VX-522, an mRNA therapy designed to treat the underlying cause of cystic fibrosis lung disease, while SpliSense is targeting a specific mutation.
Vertex Pharmaceuticals agreed to a collaboration deal with Entrada Therapeutics. The two companies announced a collaboration to develop an intracellular Endosomal Escape Vehicle.
While worthy advances have recently been made in sickle cell disease, companies such as Graphite Bio, bluebird bio, Vertex Pharmaceuticals and Editas Medicine have loftier ambitions.
The FDA had a busy week, approving drugs, greenlighting clinical trials and other regulatory activities for Immuneering, Amylyx, Sarepta, Sanofi, and Regeneron and more.
The FDA has granted a rolling review to CRISPR Therapeutics and Vertex’s exa-cel, a potential one-time treatment for sickle cell disease and transfusion-dependent beta-thalassemia.
In a recent interview with BioSpace, Imagine Pharma CEO Ngoc Thai shared the vast potential posed by a polypeptide called IMG-1 to treat Type 1 diabetes.
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