by Joseph Walker, Wall Street Journal

The gene-editing revolution is jumping from the lab to the marketplace.

The U.S. has approved the world’s first medicine employing Crispr technology, a Nobel Prize-winning discovery that promised a powerful new tool for modifying genes to treat disease and improve crop production.

The new treatment, called Casgevy and developed by Vertex Pharmaceuticals and CRISPR Therapeutics, was cleared Friday for treatment of people with the painful sickle-cell disease.

The landmark decision by the Food and Drug Administration heralds a powerful new kind of medicine, one that turns off or replaces genes to tackle conditions that have long confounded doctors and researchers.

Several companies are developing Crispr-based therapies for diseases including heart disease, cancer and rare genetic disorders. Next-generation gene-editing techniques promise to make it easier to administer the therapies with fewer side effects.

How Casgevy works for sickle-cell patients
Sickle-cell disease is caused by an inherited genetic mutation that results in a dysfunctional form of the protein, called hemoglobin, that carries oxygen in the blood.

Casgevy goes after a different gene that, when switched off, allows for the production of a form of hemoglobin that is produced when babies are in the womb that provides a functional substitute for the malformed adult hemoglobin caused by sickle-cell disease.

The therapy’s approval “shows the promise of genetic therapies that seek to treat disease at the source by making a targeted change in a person’s DNA,” said Jennifer Doudna, who shared a Nobel Prize in 2020 for her work helping discover Crispr. “It almost changes the way we define a medicine.”

Casgevy is a first step in bringing Crispr-based treatments to patients. Unlike Crispr drugs in development, it gene-edits a patient’s cells in a lab, rather than inside a patient’s body.

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