The first gene-editing treatment: 10 Breakthrough Technologies 2024


Many CRISPR treatments are in trials, but in 2022, Vertex Pharmaceuticals, based in Boston, was first to bring one to regulators for approval. That treatment was for sickle-cell. After their bone marrow was edited, nearly all the patients who volunteered in the trial were pain free. 

Good news. But the expected price tag of the gene-editing treatment is $2 to $3 million. And Vertex has no immediate plans to offer it in Africa—where sickle-cell disease is most common, and where it still kills children.

The company says this is because the treatment regimen is so complex. It involves a hospital stay; doctors remove the bone marrow, edit the cells, and then transplant them back. In countries that still struggle to cover basic health needs, the procedure remains too demanding. So simpler, cheaper ways to deliver CRISPR could come next. 


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