Forbes December 8, 2023
Topline
The Food and Drug Administration on Friday approved a new therapy based on Crispr gene editing to treat sickle cell disease, marking the first time a treatment using the technology has secured a regulatory green light in the U.S. amid hopes the revolutionary tool will emerge from the lab and transform medicine.
Key Facts
Surprising Fact
Casgevy, which edits DNA inside the human body, doesn’t actually fix the root cause of sickle cell disease. Rather than fixing the mutation responsible for faulty haemoglobin production, Casgevy targets genes responsible for producing a different kind of haemoglobin that is normally switched off shortly after birth. This genetic workaround triggers the production of typically inhibited fetal haemoglobin in the body, which helps...
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