Vertex Achieves Gene-Editing Milestone With First Ever CRISPR Therapy Approval
Great Britain is first to approve the groundbreaking CRISPR-based therapy for sickle cell and beta thalassemia. Vertex is yet to reveal its price, however, and wants to play down early expectations of its commercial potential.
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Vertex’s Casgevy has made history as the world’s first approved CRISPR gene editing therapy after the UK’s MHRA authorized the medicine ahead of its US and European regulatory counterparts.
US FDA advisory committee members questioned how potential off-target effects with Vertex’s gene therapy for sickle cell disease should be conveyed to patients and families, and whether labeling should say anything about these theoretical risks.
Having long attracted investors thanks to its promising pipeline, the moment of truth approaches for Vertex’s sickle cell gene therapy exo-cel, and VX-548, which has potential to transform acute and chronic pain treatment.