Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...
A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers ...
Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease ...
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Gene Therapy Also Effective in Younger Children With Inherited Blood Disorders
Exa-cel is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of gene editing technology through which patients' ...
ORLANDO, Dec. 6, 2025) The first study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia ...
Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label ...
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data from multiple studies demonstrating the clinical ...
US biopharma Vertex Pharmaceuticals today announced data from multiple studies demonstrating the clinical benefits of Casgevy ...
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