New data from Spherix Global Insights uncover how hematologists are defining the “right patient” for gene therapy, balancing promise with practical barriers in sickle cell disease, thalassemia, and hemophilia.
EXTON, Pa., Oct. 02, 2025 (GLOBE NEWSWIRE) -- According to the newly released Special Topix: Adoption and Impact of Gene Therapy in Hematology, physicians across sickle cell disease (SCD), beta-thalassemia, and hemophilia A and B remain firmly in a selective adoption phase. Despite the curative promise that gene therapy has long represented, actual uptake is constrained by practical realities, most prominently cost, reimbursement hurdles, conditioning regimen toxicity, and the absence of long-term safety and durability data.
Physicians consistently describe a narrow archetype of the “right” gene therapy hematologic patient: younger, treatment-motivated, and either heavily disease-burdened (as in SCD) or poorly controlled on standard care (as in thalassemia). In hemophilia, clinicians see gene therapy as more attractive for hemophilia B patients, citing stronger durability and fewer alternatives, whereas enthusiasm is muted for hemophilia A due to declining factor expression over time and robust availability of non-gene therapy options.
Brand-specific preferences are beginning to crystallize. Among hemoglobinopathies, Casgevy is emerging as the favored option over Lyfgenia and Zynteglo, with hematologists citing confidence in CRISPR/Cas9 technology and a more favorable payer outlook. In hemophilia, Hemgenix has earned more physician confidence than Roctavian, largely due to durability perceptions and lower concerns around long-term liver toxicity. Nonetheless, across all indications, referrals and actual prescribing remain limited, underscoring the chasm between clinical excitement and real-world adoption.
Importantly, the patient perspective is not fully aligned with physician readiness. In SCD particularly, physicians note frequent patient reluctance to undergo gene therapy due to the burdens of busulfan-based myeloablative conditioning and fear of the unknown. Hemophilia patients, on the other hand, are often motivated, but their physicians temper expectations by emphasizing logistical challenges and the reality that gene therapy is not always a permanent cure.
Looking ahead, hematologists anticipate that roughly one-third of their eligible patients across these conditions may ultimately receive gene therapy within the next few years, signaling a meaningful but measured integration into treatment paradigms. To accelerate adoption, stakeholders must address the tangible barriers physicians face: transparent durability data, supportive reimbursement frameworks, streamlined treatment logistics, and expanded patient education. Until then, gene therapy will remain a powerful but selectively deployed tool in the hematologist’s armamentarium.
Spherix will further explore the underlying drivers of slow adoption in an upcoming whitepaper co-authored with Dr. Steven Pipe (Medical Director of the Pediatric Hemophilia and Coagulation Disorders Program and Medical Director of the Special Coagulation Laboratory at the University of Michigan) and Dr. Haydar Frangoul (Medical Director of Pediatric Hematology/Oncology at Sarah Cannon Research Institute) offering a deep dive into physician perceptions, patient realities, and system-level hurdles intersect to shape the trajectory of gene therapy in hematology.
Special Topix™ is an independent service that includes access to a report or series of reports based on current events or topics of interest in specialty markets covered by Spherix.
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Spherix Global Insights Contacts
Sarah Hendry, Hematology Franchise Head
Sarah.hendry@spherixglobalinsights.com
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