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Editorial Advisory Board

  • Professor Andrea M. Armani, University of Southern California
  • Ruti Ben-Shlomi, Ph.D., LightSolver
  • James Butler, Ph.D., Hamamatsu
  • Natalie Fardian-Melamed, Ph.D., Columbia University
  • Justin Sigley, Ph.D., AmeriCOM
  • Professor Birgit Stiller, Max Planck Institute for the Science of Light, and Leibniz University of Hannover
  • Professor Stephen Sweeney, University of Glasgow
  • Mohan Wang, Ph.D., University of Oxford
  • Professor Xuchen Wang, Harbin Engineering University
  • Professor Stefan Witte, Delft University of Technology

Which Gene Therapy Technology Wins? New Data from Spherix Global Insights Reveals Emerging Hematologist Preferences in Beta Thalassemia Care

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. 

About Spherix Global Insights 

Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle.  

The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community.  

As a trusted advisor and industry thought leader, Spherix’s unparalleled market insights and advisory services empower clients to make better decisions and unlock opportunities for growth.  

To learn more about Spherix Global Insights, visit spherixglobalinsights.com or connect through LinkedIn.   

For more details on Spherix’s primary market research reports and interactive dashboard offerings, visit or register here: https://clientportal.spherixglobalinsights.com  

Spherix Global Insights Contacts 

Sarah Hendry, Hematology Franchise Head 

Sarah.hendry@spherixglobalinsights.com

NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight’s analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release. 


Sarah Hendry, Hematology Franchise Head
Spherix Global Insights
4848794284
sarah.hendry@spherixglobalinsights.com
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