Vertex, fresh off an R&D miss, flashes data suggesting their sickle cell CRISPR therapy is working
In one of the strongest bits of evidence yet for the effect CRISPR gene editing can have in people with genetic diseases, Vertex and CRISPR Therapeutics showed their experimental therapy kept two sickle patients from experiencing painful incidences and kept patients with another blood disorder from requiring transfusions.
The data, released today in an abstract for the American Society of Hematology, show that five patients with transfusion-dependent beta thalassemia — a disorder where patients produce insufficient hemoglobin — who received the therapy haven’t needed transfusions since the second month after administration. Previously, the patients had received between 23.5 and 61 units of blood per year.
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