National Sickle Cell Awareness Month shines a light on promising gene-editing therapy

As Sickle Cell Awareness Month this September draws attention to the urgent need for better treatments, findings from Case Western Reserve University School of Medicine researchers published in The New England Journal of Medicine show a promising gene-editing therapy that could lead to a functional cure for people living with this rare genetic blood disorder.

Led by Associate Professor Rabi Hanna, MD, and co-authored by Professor Jignesh Dalal, MD, the multicenter clinical trial found that 27 of 28 participants treated with the gene-editing therapy renizgamglogene autogedtemcel (reni-cel) had no vaso-occlusive crises following the treatment. A vaso-occlusive crisis is an extremely painful complication of sickle cell disease that occurs when sickled red blood cells block blood vessels, cutting off oxygen flow to organs and tissues.

Reni-cel uses gene editing to modify a patient’s hematopoietic stem cells—which make red blood cells—to increase levels of fetal hemoglobin. This prevents red blood cells from taking on the “crescent” shape that can block blood flow. The therapy works by collecting patients’ stem cells, modifying them in a laboratory and then infusing them back into the body after using chemotherapy to clear patients’ bone marrow.

Trial results showed that by six months, most patients’ average total hemoglobin levels had risen to near-normal levels and remained stable over time.

The publication adds to a growing body of evidence supporting gene therapy as a transformative approach to sickle cell disease, which affects about 100,000 people in the U.S. and disproportionately impacts Black Americans.

“Gene therapy is a game changer,” said Dalal, who noted that the approach marks a major advancement after more than a century of diagnosing and managing sickle cell disease without gene therapy. “For generations, patients and families living with sickle cell disease have faced tremendous challenges with remarkable resilience. Hope has always been part of their journey. Today, advances in gene-editing therapies are transforming that hope into reality, offering the possibility of a future with fewer complications, longer lives and new opportunities for patients to thrive.”

Until now, treatment options for the disease have been limited. While medications can help manage symptoms and bone marrow transplants can lead to a cure, gene-editing therapies such as reni-cel use a patient’s own cells to functionally cure the disease while avoiding the complications associated with transplants.

Researchers say the goal is not only to prevent painful crises, but also to reduce the toll sickle cell disease takes on the body over time. Repeated vaso-occlusive events can damage vital organs and shorten life expectancy. With many people diagnosed with sickle cell disease living only until their mid-40s, new and more effective therapies are especially urgent—and Case Western Reserve is playing a critical role in moving the field forward with potentially life-changing treatment options.