Bluebird Bio announces efficacy, safety HRQoL data from sickle cell studies

Bluebird bio announced new and updated efficacy, safety and health-related quality of life data from the Phase 1/2 HGB-206 Group C and Phase 3 HGB-210 studies of lovotibeglogene autotemcel gene therapy for sickle cell disease through five years of follow-up. Data were highlighted in a press briefing at the 65th American Society of Hematology Annual Meeting & Exposition. The analysis presented at ASH focused on 47 people living with sickle cell disease who received lovo-cel in the HGB-206 Group C and HGB-210 studies following enhancements to the treatment process and manufacturing protocols. Over 85% of patients required less than or equal to 2 mobilization cycles prior to infusion. As of the February 13, 2023 cutoff date, all patients had stable production of anti-sickling adult hemoglobin after infusion through last follow-up, and vaso-occlusive events and severe vaso-occlusive events were eliminated or significantly reduced in all patients, further suggesting that lovo-cel has shown a durable impact on the underlying cause of sickle cell disease. The majority of adverse events in treated patients were attributed to underlying sickle cell disease or conditioning with busulfan. The therapy was approved on December 8, 2023 by the U.S. Food and Drug Administration and is currently marketed as LYFGENIA in the U.S. Hematologic malignancy has occurred in patients treated with LYFGENIA. Monitor patients closely for evidence of mali