bluebird bio, Inc. announced that new long-term efficacy, safety and health-related quality of life (HRQoL) follow-up data from its lentiviral vector (LVV) gene therapy programs in patients with sickle cell disease who have a history of vaso-occlusive events and patients with beta-thalassemia who require regular red blood cell transfusions will be presented at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition. bluebird bio will present updated follow-up data for lovotibeglogene autotemcel (lovo-cel) in patients from the HGB-206 Group C and HGB-210 studies with sickle cell disease followed for up to 60 months (median of 35.5 months), demonstrating sustained hemoglobin AT87Q production and near-complete resolution of vaso-occlusive event (VOEs) and severe VOEs, as well as sustained improvements in HRQoL. lovo-cel treatment regimen largely reflects known side effects of hematopoietic stem cell collection and busulfan conditioning regimen and underlying sickle cell disease.

The U.S. Food and Drug Administration previously accepted the lovo-cel Biologics Licensing Application (BLA) for Priority Review and set a Prescription Drug User Fee Act (PDUFA) goal date of December 20, 2023. beti-cel was approved by the FDA in August 2022 and is commercially available in the United States as ZYNTEGLO. Once patients have the bA-T87Q-globin gene, their red blood cells (RBCs) can produce anti-sickling hemoglobin (HbAT87Q) that decreases the proportion of HbS, with the goal of reducing sickled RBCs, hemolysis, and other complications.

bluebird bio's clinical development program for lovo-cel includes the completed Phase 1/2 HGB-205 and ongoing Phase 1/2 HGB -206 and Phase 3 HGB-210 studies. bluebird bio is also conducting a long-term safety and efficacy follow-up study (LTF-307) for people who have been treated with lovo-cel in bluebird bio-sponsored clinical studies. The U.S. food and Drug Administration accepted the lovo-cel biologics Licensing Application (BLA) for Priority Review and setting a Prescription Drug User Fee act (PDUFA) goal Date of December 20, 2023).

The FDA previously granted lovo-cel orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation, and rare pediatric disease designation. These risks include, but are not limited to: may encounter additional delays in the development of programs, including the imposition of new clinical holds, which may impact ability to meet expected timelines and increase costs; the risk that the efficacy and safety results from prior and ongoing clinical trials will not continue or be seen in additional patients treated with product candidates; the risk of insertional oncogenic or other reportable patients treated with the product candidates; the risk of inserted oncogenic or other reportability.