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a cartoon image showing how CRISPR works

Two companies, Beam Therapeutics and Verve Therapeutics, are leading the charge of base editing therapies to the clinic. Last November, Beam got the green light from the US Food and Drug Administration (FDA) for its upcoming BEACON-101 trial that will use base editing to restore expression of fetal globin in bone marrow stem cells. In parallel, Verve is preparing to embark on an in vivo base editing trial to treat patients who have experienced a heart attack and have abnormally high cholesterol levels by targeting the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene, after obtaining promising results from preclinical work in rodents and non-human primates. “We’re optimistic about dosing our first patients in the second half of this year,” says Verve CEO and co-founder Sekar Kathiresan. The rapid progress of base editing programs is attracting interest from major pharmaceutical companies: in January, Beam entered a collaboration with Pfizer that could net the company up to $1.3 billion.

Beam’s rapid progress to the clinic — using technology developed just six years ago — has been built on the shoulders... see more