EXTON, PA, Aug. 14, 2025 (GLOBE NEWSWIRE) -- As the first wave of gene therapies for transfusion-dependent β-thalassemia (TDT) continues to roll out in the U.S., a new conversation is emerging within the hematology community: whether the future belongs to gene editing or gene addition.
Recent physician survey data from Market DynamixTM: Transfusion-Dependent Thalassemia reveal a clear shift in preference toward the CRISPR/Cas9-based therapy Casgevy with half of hematologists now favoring it, compared to just one-fifth for Zynteglo, a lentiviral vector-based therapy. The remaining respondents express no strong preference, citing similar efficacy and safety profiles in clinical trials.
Casgevy (exagamglogene autotemcel, Vertex/CRISPR Therapeutics) uses CRISPR/Cas9 gene editing to disable the BCL11A enhancer, reactivating the body’s natural production of fetal hemoglobin (HbF) – an oxygen-carrying molecule that can compensate for defective β-globin. “Potential for more efficient editing and promising efficacy data,” one physician noted, adding that Casgevy’s “novel CRISPR platform” and accumulating real-world experience were compelling advantages.
Zynteglo (betibeglogene autotemcel, bluebird bio) takes a different approach: inserting a functional HBB (β-globin) gene into a patient’s hematopoietic stem cells via a lentiviral vector, restoring normal hemoglobin production. Physicians highlight its “higher rate of transfusion independence (90–95% in long-term studies),” “well-established safety profile,” and “durable results” as support for their preference.
While some clinicians see Casgevy as a leap forward in precision and efficiency, others value Zynteglo’s longer track record and direct β-globin restoration. For many, the choice remains personalized, depending on patient genotype, treatment center expertise, insurance coverage, and patient or family comfort with each technology.
Despite the promise of both therapies, physicians in suburban and rural areas report that demand for gene therapy exceeds available capacity. In addition, hematologists in the south and west are more likely to cite physical distance to certified gene therapy centers as a barrier. Even when centers are accessible, high upfront costs, lengthy insurance approvals, and constrained treatment slots can delay care.
TDT is a severe inherited blood disorder requiring lifelong blood transfusions and iron chelation therapy. Gene therapy offers the potential for transfusion independence, but equitable access remains a critical challenge.
Spherix will continue to monitor the hemoglobinopathy market, including thalassemia, through annual Market DynamixTM and Patient Chart DynamixTM services.
Market Dynamix™ is an independent, data-driven service focused on understanding the evolving dynamics of specialty markets poised for disruption. Leveraging quantitative and qualitative research, the service evaluates current treatment approaches, unmet needs, and likely impact of pipeline agents over a three-to-five-year horizon.
Patient Chart Dynamix™ is an independent service that includes robust patient chart audits and integrated specialist surveys fielded biannually. This research provides an in-depth, real-world view of treatment practices by combining verified patient data with attitudinal insights from physicians. The series highlights clinical decision-making, treatment sequencing, and outcomes for targeted patient populations across key therapeutic areas.
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Spherix Global Insights Contacts
Sarah Hendry, Hematology Franchise Head
Sarah.hendry@spherixglobalinsights.com
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Sarah Hendry, Hematology Franchise Head Spherix Global Insights 4848794284 sarah.hendry@spherixglobalinsights.com
