Caring Cross, Boston Children’s collab to expand access to sickle cell gene therapy

Nonprofit organization Caring Cross and Boston Children’s Hospital 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, technology designed to ‘flip the switch’ on fetal hemoglobin production, which could provide a lifelong transformative therapy for patients with SCD. Clinical data presented at the 2025 American Society of Hematology Annual Meeting 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 collaboration aims to bring down the cost of SCD gene therapies by using Caring Cross’s decentralized place-of-care manufacturing model.