CRISPR Therapeutics and partner Vertex and bluebird bio are expected to gain US Food and Drug Administration approval for what will be two rival groundbreaking gene therapies for sickle cell disease in December – and those milestones were uppermost in the mind of Beam Therapeutics when it announced a reprioritization of its R&D strategy this week. (Also see "Vertex’s Sickle Cell Treatment Gets December Decision Date, On Track To Be First CRISPR Therapy" - Scrip, 9 June, 2023.)
The Cambridge, MA-based pioneer of base editing on 19 October announced plans to cut around 20% of its workforce (around 100 employees) and streamline its R&D ambitions in order to make faster progress with its core programs, which include two sickle cell disease candidates – ex vivo BEAM-101
Key Takeaways
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CRISPR Therapeutics/Vertex and bluebird bio could both gain US approval for their sickle cell gene therapies by the end of 2023.
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Beam Therapeutics has now made its base edited sickle cell program its priority – but its Phase I/II study will not read out until early 2025
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