Vertex Suffers Setback In Its Search For Post-CF Blockbusters
Analysts Doubt Pipeline Potential
Vertex has become a biotech investor favorite amid the tumult of 2020, but the potential of its pipeline is now under question.
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The biotech is growing fast thanks to its success in cystic fibrosis, but is under pressure to be bolder in its business development
With more data reported at ASH, Vertex and CRISPR Therapeutics added heft to the notion that functional cures are on the horizon for the inherited blood disorders sickle cell disease and beta-thalassemia. Attention is now turning to Bluebird’s marketing strategy after a filing delay.
The Phase I trial of IONIS-ENAC-2.5Rx for cystic fibrosis represents the first time an antisense medicine delivered directly to the lung has shown a significant reduction in ENaC messenger RNA levels.