KEY POINTS
  • The U.S. Food and Drug Administration has approved Casgevy, the first gene-editing treatment to be marketed in the country.
  • Casgevy uses Nobel Prize-winning technology CRISPR to treat sickle cell disease, a blood disorder that affects about 100,000 Americans.
  • Vertex Pharmaceuticals and CRISPR Therapeutics co-developed the one-time treatment that will cost $2.2 million per patient.

In this article

The U.S. Food and Drug Administration on Friday approved the country's first gene-editing treatment, Casgevy, for use in patients with sickle cell disease.

The approval comes about a decade after the discovery of CRISPR technology for editing human DNA, representing a significant scientific advancement. Yet reaching the tens of thousands of people who could benefit from the treatment could be challenging given the potential hurdles — including cost, at $2.2 million per patient — of administering the complex therapy.

Casgevy, co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics, uses Nobel Prize-winning technology CRISPR to edit a person's genes to treat disease. The treatment was approved by U.K. regulators last month.

In this article