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Vertex’s R&D Leader On Sickle Cell Challenges And ‘Out-Innovating’ Itself

David Altshuler Outlines What Sets The Biotech Apart

 
• By Andrew McConaghie

A few years ago, investors were calling on Vertex to buy in late-stage innovation, but its focus on audacious drug development goals looks set to pay off with a groundbreaking CRISPR-based sickle cell disease cell therapy approaching the market. Its R&D head David Altshuler talked to Scrip about its approach.

Vertex building logo

Vertex and its partners CRISPR Therapeutics could be just months away from achieving a two-in-one historic breakthrough: the approval of exagamglogene autotemcel (exa-cel), the first ever cell therapy for sickle cell disease and beta-thalassemia and also the first ever CRISPR gene-edited medicine.

A US Food and Drug Administration approval (a decision is expected by 8 December) would represent a major step forward in treating the life-threatening blood disorders, with early indications suggesting it could be a long-lasting functional cure for both of these inherited diseases

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