Vertex Gears Up For Major Milestones, Starting With Sickle Cell Launch And Pain Drug Readout
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.
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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.
Vertex’s Exa-Cel: Off-Target Gene Editing Analyses Sufficient Given Robust Clinical Efficacy, FDA Panel Says
Weighing Vertex’s evaluation of theoretical risks of off-target editing with the gene therapy’s strong clinical results in sickle cell disease, advisory committee says in silico and cellular analyses were sufficient but suggests closer monitoring of potential on- and off-target effects postapproval.
The Swiss pharma sees the acquisition as the ‘backbone’ for its future cardiometabolic portfolio, which could include a combination with a muscle-building therapy.