Vertex/CRISPR Prepare To Take Exa-Cel To Regulators For SCD, TDT
The first gene-edited therapy will be submitted for sickle cell disease and transfusion-dependent beta thalassemia; it will compete with bluebird in both indications, but analysts anticipate blockbuster sales in excess of $2bn.
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As the first CRISPR-based therapies draw closer to approval, the Flagship-backed biotech believes its gene writing tech is the ‘definitive’ genetic medicine platform.
The $3m gene therapy for the rare disease cerebral adrenoleukodystrophy marks bluebird’s second approval in just under a month.
The firm is laying off a big piece of its workforce and deprioritizing some investments to free up capital for potential upcoming launches, but that could add commercial risks.