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Gene Therapy Momentum Builds Among Hematologists in Sickle Cell Disease Management Despite Ongoing Access Barriers, According to Spherix Global Insights

By: via GlobeNewswire

EXTON, PA, May 01, 2025 (GLOBE NEWSWIRE) -- Patients with sickle cell disease (SCD) continue to face severe complications, despite advances in treatment that have helped mitigate some risks. Increased rates of blood clots, stroke, and other complications still significantly reduce life expectancy and impact quality of life.1 SCD, a genetic disorder affecting hemoglobin, causes red blood cells to adopt a sickle-shape, restricting oxygen flow throughout the body.2 Over the past decade, outcomes have improved with the introduction of therapies like Endari (Emmaus Life Sciences) and Adakveo (Novartis). However, progress has not been without setbacks, and substantial unmet needs remain. Now, with gene therapies Casgevy (Vertex/CRISPR Therapeutics) and Lyfgenia (bluebird bio) available for over a year, access barriers remain a major hurdle, but encouragingly, referrals for these potentially curative options are steadily increasing. 

Spherix Global Insights recently released its latest findings from Patient Chart Dynamix: Sickle Cell Disease 2025 (US), an independent chart audit analyzing 187 SCD patient charts gathered from 109 US-based hematologists. This comprehensive review reveals continued unmet need despite hematologists’ increased use of novel options, with half of patients having experienced two or more vaso-occlusive crises in the last year, and a majority reporting associated fatigue, pain, or anxiety.  

Given the significant burden SCD places on patients, hematologists are optimistic for a future with more disease-modifying therapies. One leading KOL affirms that for many of these patients, “long-term, gene therapy is the solution”. Just over half of surveyed physicians surveyed have already referred a patient for gene therapy, or in some cases, administered it themselves. However, substantial logistical hurdles remain, with the majority of physicians believing that cost will continue to be a major barrier to widespread adoption. Despite these challenges, physicians are pressing ahead, confident that most patients are strong candidates for at least a discussion about gene therapy, whether now or in the future.   

Looking ahead, new therapies – including options beyond gene therapy – are in development, promising to further shift the treatment paradigm and improve outcomes for SCD patients. More than one-third of hematologists reported awareness of products in the pipeline, with particular enthusiasm for agents such as mitapivat (Agios), inclacumab (Pfizer), and BEAM-101 (Beam Therapeutics), among others. Physicians anticipate that once these therapies become commercially available, eligible patients will be initiated on treatment relatively quickly. This optimism is, however, tempered by the persistent challenges of cost and access that have complicated prescribing and referral patterns for currently available therapies.  

This comprehensive audit is the latest in Spherix’s growing library of in-depth, data-driven insights in hematology. As the market continues to shift, and potentially curative therapies emerge, capturing the right prescriber and patient audience is paramount. Through rigorous independent research, Spherix provides this strategic insight to help pharmaceutical and biotech companies navigate the increasingly complex sickle cell disease market, reach the right patients, and ultimately improve both survival and quality of life. 

Patient Chart Dynamix™ is an independent, data-driven service unveiling real patient management patterns through rigorous analysis of large-scale patient chart audits. Insights reveal the “why” behind treatment decisions, include year over year trending to quantify key aspects of market evolution, and integrate specialists’ attitudinal & demographic data to highlight differences between stated and actual treatment patterns. 

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 

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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