The new treatment, called Casgevy and developed by Vertex Pharmaceuticals and Crispr Therapeutics, was cleared for treatment of people with the painful sickle-cell disease. Because of sickle-cell disease, Marie Tornyenu had to be hospitalized sometimes and got monthly transfusions of healthy blood from a donor. Then she realized she wasn’t feeling sickle-cell pain anymore. Sickle-cell disease, which mainly affects Black people, causes severe pain, organ damage and early death. “That the first Crispr-based therapy is in sickle-cell disease is nothing short of extraordinary," said Alexis Thompson, chief of hematology at Children’s Hospital of Philadelphia.

Source: Wall Street Journal December 09, 2023 06:34 UTC