bluebird bio to Present Long-Term Gene Therapy Data at 66th ASH Meeting

SOMERVILLE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) has revealed that fresh and updated data from its lentiviral vector (LVV) gene addition programs targeting patients suffering from sickle cell disease and beta-thalassemia will be showcased at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition. This notable event is scheduled to run from December 7-10, 2024, at the San Diego Convention Center as well as online.

Richard Colvin, M.D., Ph.D., the chief medical officer at bluebird bio, emphasized the significant long-term potential of bluebird’s gene therapies. He remarked that the company's programs are among the most thoroughly examined in the gene therapy sector, with follow-up periods extending up to a decade for patients with transfusion-dependent beta-thalassemia and almost ten years for those with sickle cell disease. Dr. Colvin expressed particular enthusiasm for the first targeted sub-analysis of lovo-cel’s clinical impact on patients with a history of stroke, which stands out among gene therapies for sickle cell disease.

bluebird bio will present updated follow-up data and analyses on lovotibeglogene autotemcel (lovo-cel) from the HGB-206 and HGB-210 studies. The findings highlight consistent clinical outcomes as early as six months post-infusion and the sustained benefits of lovo-cel. An integrated analysis focusing on sickle cell disease patients with a history of stroke will also be presented. This includes data from both overt and silent stroke cases across the HGB-206 Group A and Group C studies and the HGB-210 study, demonstrating clinical benefits in these patients and consistent outcomes within the broader lovo-cel-treated population. The safety profile of the lovo-cel treatment reflects the known effects of sickle cell disease and myeloablative conditioning, with similar safety across different age groups.

Additionally, bluebird bio will share updated long-term analyses regarding the efficacy, safety, and health-related quality of life data of betibeglogene autotemcel (beti-cel) in patients with transfusion-dependent beta-thalassemia (TDT). Data from long-term follow-up studies, extending up to ten years, show that patients with TDT achieved durable transfusion independence and normal or near-normal hemoglobin levels, regardless of their genotype and age, while maintaining a favorable long-term safety profile.

The presentations include:

1. Oral Presentation [#511]: An Update on Lovotibeglogene Autotemcel (lovo-cel) Clinical Trials for Sickle Cell Disease (SCD) and Analysis of Early Predictors of Response to Lovo-cel. Presenter: Dr. Stacey Rifkin-Zenenberg. Date/Time: Sunday, December 8, 2024, 9:30 a.m. – 11:00 a.m. PT.

2. Poster Presentation [#3576]: Participants with a History of Stroke in Lovotibeglogene Autotemcel (lovo-cel) Clinical Trials. Presenter: Dr. Jen Jaroscak. Date/Time: Sunday, December 8, 2024, 6:00 p.m. – 8:00 p.m. PT.

3. Poster Presentation [#2194]: Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy results in durable Hemoglobin A (HbA) Production with up to 10 Years of Follow-Up in Participants with Transfusion-Dependent β-Thalassemia. Presenter: Dr. Alexis A Thompson. Date/Time: Saturday, December 7, 2024, 5:30 p.m. – 7:30 p.m. PT.

Lovo-cel received U.S. Food and Drug Administration (FDA) approval in December 2023 and is commercially available in the United States as LYFGENIA. Similarly, beti-cel was approved by the FDA in August 2022 and is available in the U.S. under the name ZYNTEGLO.

About LYFGENIA™ (lovotibeglogene autotemcel):
LYFGENIA is a one-time ex-vivo lentiviral vector gene therapy approved for treating patients aged 12 and older with sickle cell disease and a history of vaso-occlusive events (VOEs). It functions by adding a functional β-globin gene to the patients’ hematopoietic stem cells (HSCs), enabling the production of adult hemoglobin with anti-sickling properties (HbAT87Q), which can reduce VOEs. The safety and efficacy of LYFGENIA are being evaluated in the ongoing Phase 3 HGB-210 study, with long-term follow-up studies also underway.

About ZYNTEGLO™ (betibeglogene autotemcel):
ZYNTEGLO is a pioneering one-time ex-vivo LVV gene therapy for adults and pediatric patients requiring regular red blood cell transfusions due to beta-thalassemia. It works by inserting functional copies of a modified β-globin gene into a patient’s stem cells, subsequently producing modified functional adult hemoglobin (HbAT87Q). This can potentially elevate hemoglobin levels to normal or near-normal, eliminating the need for regular transfusions.