New cell-based gene therapies provide hope for sickle cell disease patients

By Stacy M. BrownNNPA Newswire Senior National Correspondent@StacyBrownMedia The U.S. Food and Drug Administration (FDA) has greenlit two revolutionary cell-based gene therapies, Casgevy and Lyfgenia, marking a significant leap forward in treating sickle cell disease (SCD) for patients aged 12 and older. The approval by the FDA signifies the commencement of a novel epoch in managing sickle cell disease, providing optimism to individuals whose lives have been significantly disrupted by the arduous condition. Sickle cell disease, a group of inherited blood disorders, affects around 100,000 individuals in the United States and is predominant among African Americans. Health officials said the root cause of SCD is a mutation affecting hemoglobin, a crucial protein in red blood cells responsible for oxygen delivery. The genetic problem causes red blood cells to have a unique “sickle” shape, which can lead to vaso-occlusive events (VOEs) or vaso-occlusive crises (VOCs), which are very painful and damage organs. The recurrence of these crises poses life-threatening risks and potential disabilities. “Sickle cell disease is a rare, debilitating, and life-threatening blood disorder with significant unmet need, and we are excited to advance the field,” said Nicole Verdun, M.D., director of the Office of Therapeutic Products within the FDA’s Center...