Can Beam Keep Up With Sickle Cell Gene Therapy Frontrunners? Investors Are Unconvinced

Companies aiming to be fast followers in sickle cell disease gene therapy behind Vertex and bluebird have thinned out in 2023, and Beam may struggle to convince investors its base editing approach can compete.

Beam Therapeutics

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.)

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