New partnership aims to expand global access to sickle cell gene therapy

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 Orlando. 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. 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."

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

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."