bluebird bio Completes First Cell Collection for LYFGENIA™ Gene Therapy

In a landmark achievement, bluebird bio, Inc. (Nasdaq: BLUE) has successfully completed the first commercial cell collection for LYFGENIA (lovotibeglogene autotemcel), a gene therapy designed to treat sickle cell disease (SCD) in patients who have experienced vaso-occlusive events. Approved by the FDA in December 2023, LYFGENIA stands as the most thoroughly researched gene therapy for SCD, featuring the most extended follow-up periods in the field.

Andrew Obenshain, president and CEO of bluebird bio, expressed his enthusiasm, stating, "Seeing people living with sickle cell disease receive gene therapy in the real world is a vision that has fueled bluebird for more than 10 years." He emphasized the historical significance of this milestone, which comes nearly a century after sickle cell disease was first identified at the molecular level and almost a decade after bluebird began clinical development for LYFGENIA. He extended gratitude to the patients, caregivers, researchers, and clinicians who contributed to this achievement and anticipated ongoing collaboration with the SCD community.

The inaugural cell collection occurred at Children’s National Hospital in Washington, DC, a member of bluebird’s network of over 60 Qualified Treatment Centers (QTCs) across the nation. These centers are chosen based on their expertise in transplant, cell and gene therapy, and sickle cell disease, receiving specialized training to administer LYFGENIA.

David Jacobsohn, MD, MBA, chief of the division of Blood and Marrow Transplantation at Children’s National Hospital, also shared his excitement, stating, "We are thrilled to be the first center in the country to commercially collect cells from a person living with sickle cell disease and are proud to be the trailblazers in using this new approach." He highlighted the approval of gene therapies for SCD as a monumental breakthrough in patient care, offering families hope amidst their struggles.

LYFGENIA is intended for patients aged 12 and older with SCD and a history of vaso-occlusive events. It operates by introducing a functional β-globin gene into the patients' hematopoietic stem cells (HSCs), enhancing the production of adult hemoglobin with anti-sickling properties (HbAT87Q). The therapy has shown potential in reducing vaso-occlusive events and has been evaluated in comprehensive clinical studies, including the complete Phase 1/2 HGB-206 study and the ongoing Phase 3 HGB-210 study. bluebird bio is also conducting a long-term follow-up study (LTF-307) to assess the safety and efficacy of LYFGENIA.

However, LYFGENIA is not without limitations and safety concerns. Patients with α-thalassemia trait may require chronic red blood cell transfusions due to anemia with erythroid dysplasia. The therapy has not been studied in individuals with more than two α-globin gene deletions. A boxed warning highlights the risk of hematologic malignancies, necessitating lifelong monitoring of patients. Other risks include delayed platelet engraftment, neutrophil engraftment failure, insertional oncogenesis, and hypersensitivity reactions. There are also specific guidelines regarding the use of anti-retroviral medications, hydroxyurea, and iron chelation in conjunction with LYFGENIA.

bluebird bio, founded in 2010, has established itself as a leader in gene therapy, securing FDA approvals for three therapies in under two years. The company focuses on severe genetic diseases and has developed a robust dataset for ex-vivo gene therapies. bluebird bio remains dedicated to advancing the field of gene therapy and supporting patient communities through innovative solutions and a people-centric culture.