Companies team up to support sickle cell gene therapy Lyfgenia
Xcellbio technology to be used along with Bluebird assay
Bluebird Bio and Xcellbio have entered into an agreement to support the commercialization of Lyfgenia (lovotibeglogene autotemcel), Bluebird’s gene therapy for sickle cell disease, incorporating Xcellbio technology into Bluebird’s potency assay with the aim of helping to ensure the therapy’s effectiveness.
“We’re humbled to play a small part in what is a transformative treatment option for patients suffering from sickle cell disease,” Brian Feth, CEO of Xcellbio, said in a company press release. “This agreement advances our business strategy of supporting new modalities in order to bring transformative treatment options to patients.”
Sickle cell disease is caused by mutations that lead to the production of an abnormal form of hemoglobin, the protein that red blood cells use to transport oxygen through the body. The abnormal protein is prone to clumping inside red blood cells, deforming them into the sickle-like shape that gives the disease its name. The deformed cells tend to get trapped inside blood vessels, restricting or blocking blood flow. Blood vessel obstruction and blood flow disruption can cause vaso-occlusive crises, acute chest syndrome, and other complications.
Sickle cell gene therapy
Lyfgenia treatment involves collecting blood stem cells from a patient’s bone marrow and engineering them to enable the production of a version of hemoglobin with anti-sickling properties. The genetically modified stem cells are returned to the patient via a stem cell transplant. This allows the production of red blood cells that are resistant to sickling, which in turn is expected to reduce the frequency of vaso-occlusive crises and other sickle cell complications.
Lyfgenia is approved in the U.S. to treat sickle cell patients, ages 12 and older, who have a history of vaso-occlusive events.
Under the agreement, Xcellbio’s cell incubation technology will be incorporated into tests Bluebird uses to assess the modified stem cells before they are returned to patients and to predict Lyfgenia’s effectiveness. According to Xcellbio, the technology aims to replicate the environments in the body where red blood cell sickling normally occurs.
By testing the modified cells in these artificial environments before they are returned to patients, the assay aims to ensure that patients will receive stem cells that can produce red blood cells capable of resisting sickling so that the gene therapy has the best chance of providing benefits.
The collaboration leverages Xcellbio’s proprietary technology platform known as AVATAR and an incubation technology called HYPERPRESS.
Financial terms of the collaboration were not disclosed.
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