New Study Shows CRISPR Gene Therapy Fights Sickle Cell

On Tuesday, June 18, 2024, new developments in the fight against sickle cell disease emerged as a recent trial showcased the effectiveness of CRISPR gene-editing therapy for patients. Dr. Rabi Hanna, the lead investigator of the study, has expressed optimism about the treatment's potential. Dr. Hanna is the chair of pediatric hematology oncology and blood and marrow transplantation at Cleveland Clinic Children’s.

Sickle cell anemia is a hereditary condition that causes red blood cells to become misshapen and sickle-shaped, leading to severe pain and other complications. The disease affects approximately 100,000 Americans, predominantly among Black Americans, and can significantly reduce life expectancy.

The Cleveland Clinic has been experimenting with a CRISPR-based therapy known as renizgamglogene autogedtemcel, or "reni-cel." This one-time treatment involves using the patient's own stem cells to address the genetic defect responsible for sickle cell disease.

In the latest trial, 18 patients underwent this experimental treatment, with two of them being treated at the Cleveland Clinic. The procedure involved harvesting patients' stem cells, which were then genetically edited in the lab. Patients also received chemotherapy to prepare their bone marrow for the infusion of the modified stem cells.

According to a news release from the clinic, all patients in the trial successfully recovered their white blood cells and platelets after the treatment. Additionally, none of the patients experienced painful episodes post-treatment, and those monitored for more than five months showed a resolution of their anemia.

The absence of serious side effects further underscores the promise of this innovative therapy. Dr. Hanna emphasized that these positive results bring hope for a functional cure for this debilitating disease.

The findings were shared at the European Hematology Association 2024 Hybrid Congress (EHA) in Madrid. It is important to note that these results are considered preliminary until they are published in a peer-reviewed journal.

The trial received funding from Editas Medicine, a company specializing in gene editing.