(Press-News.org) Gaithersburg, Maryland and Boston, MA — January 20, 2026 — Caring Cross, a 501(c)(3) non-profit dedicated to improving access to advanced therapies, and Boston Children’s Hospital today announced a collaboration to provide a sustainable, affordable pathway for patients to access stem cell gene therapies for the treatment of Sickle Cell Disease (SCD).
The partnership centers on a worldwide license granted to Caring Cross by Boston Children’s for lentiviral-based BCL11A-LCRshRNAmiR, an innovative technology designed to “flip the switch” on fetal hemoglobin production. This provides a potential lifelong transformative therapy for patients with SCD. The lentiviral vector was developed in Dr. David Williams’ laboratory based on fundamental research in Dr. Stuart Orkin’s laboratory at Boston Children’s Hospital.
Bridging the Gap Between Innovation and Access
While currently approved gene therapies for SCD and BT have demonstrated outstanding results in mitigating the burden of these diseases, their current price tag – which can exceed $3 million per dose – places them out of reach for the vast majority of the world’s patients.
This collaboration seeks to bridge that gap by utilizing Caring Cross’s decentralized “place-of-care” manufacturing model, which aims to deliver these life-saving treatments at a fraction of their current market cost.
Clinically Validated Success
The announcement follows the presentation of groundbreaking clinical data at the 2025 American Society of Hematology (ASH) Annual Meeting in Orlando1. The data demonstrated that SCD patients treated with the BCL11A shmiR-based therapy showed robust, durable increases in fetal hemoglobin with sustained mitigation of SCD disease manifestations.
“The data shared at ASH 2025 and the results of the ongoing phase 2 GRASP trial confirm and extend previous findings that the therapy is safe and effective,” said Dr. David A. Williams, Chief, Division of Hematology/Oncology, Boston Children’s Hospital, President, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, and Leland Fikes Professor of Pediatrics at Harvard Medical School. “Our next goal is to complete data collection of the pivotal phase II clinical trial (NCT05353647) and then ensure that this therapy reaches patients around the world, particularly those who are marginalized and underserved. By collaborating with Caring Cross, we are taking a definitive step toward making this therapy accessible to many more people that suffer from SCD and potentially Beta Thalassemia.”
A Global Mission for Health Equity
This collaboration expands access beyond the USA to regions bearing the heaviest burden of SCD, including sub-Saharan Africa, India, the Middle East, and the Global South. By partnering with local hospitals and non-profits, the initiative is creating a scalable blueprint to democratize cellular gene therapies and establish them as global standard of care.
“This agreement is an important milestone in our effort to improve access to cellular gene therapies,” said Dr. Boro Dropulić, Founder and Executive Director of Caring Cross. “By securing the rights to BCL11A shmiR technology, we empower hospitals currently performing bone marrow transplantation to expand their capabilities and produce gene-modified stem cells for the treatment of SCD and BT. Our sustainable access model will ensure that these advancements remain economically viable for healthcare systems and life-changing for the patients who need them most.”
https://www.sciencedirect.com/science/article/pii/S0006497125052802
Upcoming Virtual Seminar
Caring Cross will host a virtual seminar featuring a live presentation by Dr. David A. Williams on the clinical results of the BCL11A shmiR trial and the pathway to global affordability.
Title: Democratizing Access to Cellular Gene Therapies: SCD Gene Therapy Clinical Updates and the Non-Profit Pathway for Global Accessibility
Date: January 29, 2026
Time: 3:00 PM EST
Registration: https://caringcross.org/events/
About Caring Cross
Caring Cross is a 501(c)(3) nonprofit dedicated to accelerating the development of and ensuring global access to advanced therapies. To achieve its mission, Caring Cross develops technologies and therapeutic candidates that improve the accessibility and affordability of advanced medicines like CAR-T therapy and stem cell gene therapy. Lentiviral vectors used to manufacture Caring Cross advanced therapy medicinal products are manufactured by Vector BioMed, a for-profit contract development and manufacturing organization (CDMO) specializing in rapid manufacturing solutions to provide the industry with affordable, high-quality GMP lentiviral vectors.
For more information on Caring Cross, visit https://caringcross.org
For more information on Vector BioMed, visit https://vectorbiomed.com
Media Contact:
Daniel Laender
Director of Communications
daniel.laender@caringcross.org
+1 (240) 447-3692
About Boston Children’s Hospital
Boston Children’s Hospital is ranked among the best children’s hospitals in the nation by U.S. News & World Report and is a pediatric teaching affiliate of Harvard Medical School. Home to the world’s largest research enterprise based at a pediatric medical center, Boston Children’s has led the way in life-changing pediatric innovation since its founding in 1869. Today, 3,000 researchers and scientific staff, including 14 members of the National Academy of Sciences, 37 members of the National Academy of Medicine and 13 Howard Hughes Medical Investigators across ~1M square feet of lab space comprise the research community. From bench to bedside, scientists work on preventing, treating, and curing diseases that impact both children and adults, no matter how rare or complex the condition. Founded as a 20-bed hospital for children, Boston Children’s is now a 491-bed comprehensive center for pediatric and adolescent health care.
With nine satellite locations and the Martha Elliot Health Center, Boston Children’s also provides 24/7 pediatric care at five hospitals including Beverly Hospital, Winchester Hospital, St. Luke’s Hospital, South Shore Hospital and Cape Cod Hospital.
Boston Children’s also includes: Affiliation with Franciscan Children’s Hospital, including 112 beds and 700+ employees; Boston Children’s Primary Care Alliance, a robust network of 33 pediatric practices serving patients and families throughout Massachusetts; The Pediatric Physicians’ Organization at Boston Children’s Hospital (PPOC) with more than 400 physicians, nurse practitioners, and physician assistants devoted exclusively to pediatric primary care, in close collaboration with subspecialists at Boston Children’s in more than 90 locations throughout Massachusetts; Boston Children’s Health Physicians (BCHP) which is the largest pediatric multispecialty group in New York’s Metropolitan Area, the Hudson Valley, and Connecticut, including nearly 300 clinicians in more than 60 locations.
For more, visit our Answers blog and follow us on Facebook, YouTube, and LinkedIn.
Media Contact:
Joelle Zaslow
Joelle.zaslow@childrens.harvard.edu
END
Caring Cross and Boston Children’s Hospital collaborate to expand access to gene therapy for sickle cell disease and beta thalassemia
2026-01-22
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