In race for in vivo sickle cell cure, Intellia unveils (very) early proof-of-concept
Vertex and CRISPR Therapeutics have shown they can use the gene editing tool to cure, for at least a couple years, a handful of patients with sickle cell disease and are now entering late-stage development.
It’s a major advance, but it comes with the same caveats that have plagued other gene therapies for sickle cell. You have to give patients what amounts to a bone marrow transplant: a laborious, expensive and occasionally risky procedure that involves removing stem cells from their marrow, editing them in a facility and then re-injecting them. Any company that could find a way to edit cells with a simple IV infusion could make those older approaches obsolete.
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