bluebird bio Announces Completion of First Cell Collection for LYFGENIA Gene Therapy

bluebird bio, Inc. (Nasdaq: BLUE) announced today that it has completed the first commercial cell collection for LYFGENIA (lovotibeglogene autotemcel), a one-time gene therapy for the treatment of sickle cell disease in patients with a history of vaso-occlusive events. LYFGENIA was approved by the FDA in December 2023 and is the most deeply studied gene therapy for sickle cell disease, with the longest follow-up in the field.

“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,” said Andrew Obenshain, president and CEO, bluebird bio. “This historic moment comes nearly a century after sickle cell disease was the first genetic disorder to be characterized at the molecular level, and almost a decade after bluebird initiated clinical development for LYFGENIA. We are grateful to the patients, caregivers, researchers, and clinicians whose work made this milestone possible, and look forward to continued partnership with the sickle cell disease community.”

The patient’s cells were collected at Children’s National Hospital in Washington, DC, which is part of bluebird’s national network of more than 60 Qualified Treatment Centers (QTCs). QTCs are selected based on leading expertise in transplant, cell and gene therapy, and sickle cell disease and receive specialized training to administer LYFGENIA.

“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. The recent approval of gene therapies to treat patients with sickle cell is an enormous breakthrough in patient care and a silver lining to families witnessing their children’s struggles with this condition. In the face of immense burden, gene therapy is a groundbreaking treatment alternative and a beacon of hope for a better future,” said David Jacobsohn, MD, MBA, chief of the division of Blood and Marrow Transplantation at Children’s National Hospital.

About LYFGENIA (lovotibeglogene autotemcel) or lovo-cel

LYFGENIA is a one-time ex-vivo lentiviral vector gene therapy approved for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events (VOEs). LYFGENIA works by adding a functional β-globin gene to patients’ own hematopoietic (blood) stem cells (HSCs). Durable production of adult hemoglobin with anti-sickling properties (HbAT87Q) is possible following successful engraftment. HbAT87Q has a similar oxygen-binding affinity to wild-type HbA, limits sickling of red blood cells and has the potential to reduce VOEs.