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With 1 Cure Behind It, This Breakthrough Biotech Takes Aim at a Bigger Market

With 1 Cure Behind It, This Breakthrough Biotech Takes Aim at a Bigger Market

Long before they won the 2020 Nobel Prize in Chemistry for discovering the molecular gene-editing tool known as clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9, Jennifer Doudna and Emmanuelle Charpentier dreamed of Victoria Gray, a woman with sickle cell disease. Although they didn't know her, they knew that one day, a patient with a genetic disease would be cured using their discovery. Sickle cell disease was the logical place to start since it's the most common blood disorder with a single-gene mutation.