Beam Reports Positive Pivotal Results for Sickle Cell Gene-Editing Therapy

Rare Daily Staff

Beam Therapeutics said Tuesday that its experimental sickle cell disease therapy, built on a newer and more precise form of gene editing, met its main goal in a pivotal trial: keeping participants free of severe pain crises for at least a year.

The company said the treatment, called risto-cel, also met all of the trial’s safety goals in a planned early analysis. It met every secondary goal as well, including keeping patients out of the hospital for severe crises for at least 12 straight months. Independent reviewers assessed the results.

Beam plans to ask the U.S. Food and Drug Administration for approval as early as the end of 2026. That would be the first approval application for a therapy based on base editing, a technique that changes a single letter of DNA without cutting through both strands of the DNA molecule.

Beam will present data from an earlier cutoff at the American Society of Hematology annual meeting in New Orleans in December. Full results will not be presented until a medical meeting in 2027.

Sickle cell disease is caused by a single error in a gene that makes part of hemoglobin, the protein that carries oxygen in red blood cells. The error makes red blood cells stiff and crescent-shaped. These cells can block blood vessels and cause episodes of severe pain, known as vaso-occlusive crises.

Risto-cel doesn’t fix that error directly. Instead, it switches back on fetal hemoglobin, a form of hemoglobin that everyone makes before birth and that doesn’t sickle. The body normally stops making fetal hemoglobin in infancy. Doctors collect a patient’s own blood stem cells, and Beam edits them so a protein that normally shuts off fetal hemoglobin can no longer do so. The edit mimics a natural genetic variation: some people carry it and keep making fetal hemoglobin all their lives.

“For patients with SCD, severe vaso-occlusive crises can be debilitating and life-threatening, making sustained freedom from these events an important clinical outcome,” said Beam CEO John Evans.