Lovotibeglogene autotemcel, under the brand name Lyfgenia, is a lentiviral gene therapy used for the treatment of sickle cell disease.
Routes of administration Intravenous.
The most common side effects include stomatitis, low white blood cells, thrombocytopenia and anemia, and febrile neutropenia consistent with chemotherapy and underlying disease.
Lovotibeglogene autotemcel is indicated for the treatment of people aged twelve years of age and older with sickle cell disease and a history of vaso-occlusive events.
Lyfgenia is a cell-based gene therapy.
Lyfgenia uses a lentiviral vector as a gene delivery vehicle for genetic modification and is approved for the treatment of patients 12 years of age and older with sickle cell disease and a history of vaso-occlusive events.
WithLovotibeglogene autotemcel, the patient’s blood stem cells are genetically modified to produce HbAT87Q, a gene-therapy derived hemoglobin that functions similarly to hemoglobin A, which is the normal adult hemoglobin produced in persons not affected by sickle cell disease.
Red blood cells containing HbAT87Q have a lower risk of sickling and occluding blood flow.
These modified stem cells are then delivered to the patient.
The recipient’s blood stem cells are genetically modified to produce HbA a gene-therapy derived hemoglobin A, which is similar to the normal adult hemoglobin produced in persons not affected by sickle cell disease.
These modified stem cells are then delivered to the recipient.
The gene therapy is made from the recipient’s own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion as part of a hematopoietic stem cell transplant.
Prior to treatment, the recipient’s own stem cells are collected, and then the recipient must undergo myeloablative conditioning with high-dose chemotherapy, a process that removes cells from the bone marrow so they can be replaced with the modified cells in lovotibeglogene autotemcel.
Twenty-eight (88%) of 32 participants achieved vaso occlusive episode relief during this time period.
The cost effectiveness threshold of the therapy is estimated to be between US$1.35 million to $2.05 million.