The FDA’s approval of Casgevy, which uses the Nobel Prize-winning gene-editing therapy to treat patients with sicke cell disease, opens the door to many other therapies.
Updated, Dec. 8: The Food and Drug Administration on Friday approved the first drug to treat patients with sickle cell disease using the Nobel Prize-winning CRISPR gene-editing therapy. Casgevy (also known as Exa-cel) is manufactured by Vertex Pharmaceuticals in Boston and CRISPR Therapeutics in Zug, Switzerland. The FDA also approved a conventional sickle cell disease treatment called Lyfgenia, by Bluebird Bio in Somerville, Massachusetts. This article was originally published on Dec. 5 and updated after news of the FDA’s announcement.
Victoria Gray never remembers a moment when she didn’t have sickle cell disease. She was diagnosed with the condition at 3 months of age after she cried so hard during a bath that she was taken to the emergency room. For Gray, who has no close relatives with the disorder, that was the beginning of unrelenting pain, ER visits, hospitalizations and treatments that robbed her of much of her childhood and adult life.
Gray became unable to take care of herself and her family. In hopes that she could improve her life by even 50%, Gray bravely chose to be the first patient to enter a clinical trial using a CRISPR editing drug to treat sickle cell disease. Four and a half years later she, and dozens of others from the trial, are doing amazingly well.
The first marketed medicine based on gene-editing CRISPR (clustered regularly interspaced short palindromic repeats) technology, called Exa-cel, is expected to receive approval from the Food and Drug Administration this week for use in selected patients with sickle cell disease. Exa-cel recently received approval in the U.K. for treatment of both sickle cell and beta-thalassemia.
“I never imagined I could feel this well and do so much,” Gray said on a recent Zoom call, after we first met at a Concert Genetics meeting in Nashville in October.
Sickle cell disease is a lifelong, inherited condition that affects hemoglobin, a protein that carries oxygen throughout the body in red blood cells. Normal red blood cells are disc-shaped and flexible enough to move easily throughout the blood vessels. But in people who inherit two mutations for sickle cell disease, abnormal hemoglobin forms and causes the red blood cells to become sickle-shaped, jamming blood flow and often leading to pain and serious health problems.
Sickle cell can occur in people of any ancestry, but it’s most common in those who identify as Black.
