FDA approval: Lyfgenia (from Catapult’s regulatory round-up – December 2023)

FDA has granted BLA approval for Lyfgenia (lovotibeglogene autotemcel), a cell-based gene therapy for patients ages 12 and older with sickle cell disease and a history of vaso-occlusive events. Lyfgenia uses a lentiviral vector (gene delivery vehicle) for genetic modification. With Lyfgenia, 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.

Published on the 5th of January 2024. Read the entire regulatory round-up for December 2023 here.