Vertex Pharmaceuticals (VRTX US), the global leader in cystic fibrosis treatment, has a broad and deep clinical pipeline across multiple modalities. Each of its clinical programs represents a multibillion-dollar opportunity.
MRNA drug candidate for treating the remaining 5,000 CF patients who cannot benefit from existing therapies, is set to enter clinic. Drug candidate for acute pain is in late-stage trial.
Regulatory submission for exa-cel for sickle cell disease and transfusion-dependent beta thalassemia in U.S., EU and UK are expected to be completed by 1Q23.
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