Beam says its gene-editing treatment for sickle cell disease met its main goal in a key trial and plans to seek FDA approval
Beam released only top-line results, without the numbers. If approved, it would be the first medicine to use a precise kind of gene editing called base editing.
A one-time gene-editing treatment for sickle cell disease met its main goal in the trial meant to support its approval, its maker, Beam Therapeutics, said Nov. 10. The company plans to apply for approval from the U.S. Food and Drug Administration as early as the end of 2026.
Sickle cell disease is an inherited blood disorder that affects about 100,000 people in the U.S., most of them African American, according to the FDA. A single error in the gene for hemoglobin, the protein in red blood cells that carries oxygen, makes the cells bend into a stiff crescent, or sickle, shape. Those cells clog blood vessels, causing bouts of severe pain called vaso-occlusive crises, organ damage and early death.
How the treatment works
Risto-cel uses base editing, a newer form of gene editing that changes a single letter of DNA without cutting both strands of the DNA, as older CRISPR tools do, Beam says. Doctors collect a patient’s own blood stem cells, Beam edits them in a lab, and the cells are returned in a stem cell transplant. The edit is meant to switch back on fetal hemoglobin, a form of hemoglobin made before birth that does not sickle. Before the transplant, patients get high-dose chemotherapy with a drug called busulfan to clear room in the bone marrow.
What the trial showed
The trial, called BEACON, had treated 39 adults and 10 teenagers as of Oct. 20. Of the 31 patients followed long enough, the treatment met the main goal: no severe pain crises for at least 12 months in a row, as judged by independent reviewers, Beam says. It also met its goals for avoiding hospital stays for crises and for safety. Beam did not say how many patients reached the goal. Side effects matched those expected from the cell collection, chemotherapy and transplant, the company says. Detailed results are planned for a medical meeting in 2027. The trial had no comparison group.
Existing options
The FDA approved the first two gene therapies for sickle cell disease in December 2023: Casgevy, which uses CRISPR to raise fetal hemoglobin, and Lyfgenia, which adds a working hemoglobin gene. Both also require chemotherapy and a stem cell transplant. In Casgevy’s key trial, 29 of 31 evaluable patients went at least 12 months without a severe crisis, the FDA said then. Risto-cel is not approved, and Beam did not discuss price.
Source: Beam Therapeutics announcement via GlobeNewswire, Nov. 10, 2026 and FDA announcement of the first sickle cell gene therapies, Dec. 8, 2023.