Bluebird Bio presents long-term data on LYFGENIA gene therapy for SCD

Bluebird Bio (BLUE) announced new and updated data from LYFGENIA gene therapy for patients with sickle cell disease who have a history of vaso-occlusive events. As of July 2024, 70 patients were treated across the full lovo-cel clinical development program, with follow-up beyond 9 years in the earliest treated patients. Updated efficacy data continue to support sustained, transformational impact on VOE burden and hematologic markers of disease. As of the July 2024 cutoff date, all patients continued to have stable production of anti-sickling adult hemoglobin after infusion through last follow-up and total Hb at last visit was 12.4 g/dL and was stable without transfusion support post engraftment. VOEs and severe vaso-occlusive events were eliminated or significantly reduced in all patients. Specific findings include: 36/38 of evaluable patients achieved complete resolution of severe VOEs in the 6-18 months post infusion, maintained for a median of 42.3 months; 33/38 of evaluable patients achieved complete resolution of VOEs, maintained for a median of 42.4 months; 10/10 pediatric patients achieved complete resolution of VOEs and sVOEs. The safety profile of the lovo-cel treatment regimen was generally consistent with underlying sickle cell disease and the known effects of myeloablative conditioning. There were no cases of graft failure or graft-versus-host disease, no vector-related complications, and no insertional oncogenesis. For complete safety information please refer to the U.S. Prescribing Information noted below. Data from patients with sickle cell disease and a history of overt stroke show no recurrence of stroke following treatment with lovo-cel. Data showed that patients with a history of overt stroke remained stable without recurrent stroke up to 9 years post-treatment, with median follow-up of 6.5 years.