Rabi Hanna, MD, on Practical Considerations for Integrating Gene Therapy

“I cannot emphasize how much misinformation there is on gene therapy. Many patients come thinking it is just an injection or infusion that they get in the clinic.”

Since the FDA’s late 2023 approval of Vertex Pharmaceuticals' and CRISPR Therapeutics’ gene editing therapy exagamglogene autotemcel (exa-cel; marketed as Casgevy) and bluebird bio’s gene addition therapy lovotibeglogene autotemcel (lovo-cel; marketed as Lyfgenia) for sickle cell disease (SCD), these products have begun to be rolled out to clinical treatment centers in the United States. As institutions begin to use these products for the first time in the commercial setting, there are a number of important factors that providers and other stakeholders need to consider.

At the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 6 to 9, 2025, in Orlando, Florida, CGTLive® sat down with Rabi Hanna, MD, the chairman of the Division of Pediatric Hematology & Oncology and BMT at Cleveland Clinic Children's, whose institution recently began offering treatment with exa-cel and lovo-cel to patients with SCD in the commercial setting, to learn more. Hanna discussed what it takes for a center to responsibly offer gene therapy for SCD and how the Cleveland Clinic prepared for that step. He emphasized that gene therapy cannot exist in isolation. Programs must first build a comprehensive sickle cell infrastructure that includes other disease modifying treatments, pain management, and strong psychosocial support. Gene therapy is only one option within a broader care model aimed at addressing decades of health disparities affecting this patient population.

Hanna also stressed the importance of careful patient education, noting widespread misconceptions about gene therapy. He said that patients often expect a simple infusion, so the team schedules multiple consultation visits that include nurses and social workers to explain the long, complex process and its uncertainties, particularly around stem cell collection and manufacturing.

Hanna also discussed the importance of long-term follow up and safety evaluation, pointing out that patients who were treated in the clinical trials for SCD gene therapy will continue to be monitored for 15 years after their initial treatment. Lastly, Hanna described how gene therapy in general has expanded interdisciplinary collaboration, bringing together specialists across genetics, hematology, cardiology, gastroenterology, and other fields as gene-based treatments move into more diseases.

For more coverage of ASH 2025, click here.