Robin E. Miller, MD, on Integrating Gene Therapy into Clinical Care for Sickle Cell Disease

“It's a very new therapy and the press has been very positive, and so families often don't understand how difficult the process is going to be. As such, we need to explore all the options that the patients have. Some of them have already failed disease modifying therapies and are really looking for something else, but some haven't tried disease modifying therapies yet, and we might direct them that way first.”

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) were approved by the FDA for the treatment of sickle cell disease (SCD) in late 2023. Since then, these products have begun to be adopted as part of standard care by some clinics across the United States. Although, because of the complexity of these therapies, clinical practices need to consider a number of key factors and make important preparations before they begin offering these treatments.

At the the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 6 to 9, 2025, in Orlando, Florida, CGTLive® spoke with Robin E. Miller, MD, the director of the Sickle Cell Disease Program at Nemours Children’s Hospital, to learn more about the practical considerations for treatment centers that are integrating or considering integrating gene therapy into their clinical care for SCD. Miller stressed that centers need key infrastructure, including SCD specialists, transplant experts, a robust blood bank, a stem cell lab, and experience with bone marrow transplant in SCD. Also important are psychosocial supports, including psychologists and social workers familiar with the unique challenges that families of patients with SCD face.

She also explained that determining candidacy requires multidisciplinary input and shared decision making. Families must understand that while gene therapy is promising, it is a long process and carries risks. Some patients may be better suited for other therapies or traditional stem cell transplant, especially if a matched donor is available. Miller emphasized the importance of clearly explaining risks, benefits, and practical factors like recovery time and long-term follow-up.

For more coverage of ASH 2025, click here.