The FDA has approved a gene-edited treatment for sickle cell disease, marking a significant medical advancement.
The treatment, known as CASEVY, utilizes CRISPR technology to edit genes and target the BCL11A gene, allowing continued production of fetal hemoglobin and reducing damage from sickle cells.
While it may not cure the disease, it has the potential to eliminate most symptoms. (Trending: Hunter Biden’s Own Memoir Is Coming Back To Haunt Him In Criminal Trial)
Patients will undergo a bone marrow transplant and receive chemotherapy, with long-term effectiveness and potential adverse effects still under investigation.
Reshma Kewalramani, M.D., chief executive officer and president of Vertex Pharmaceuticals wrote, “CASGEVY’s approval by the FDA is momentous,” in a statement.
Adding, “It is the first CRISPR-based gene-editing therapy to be approved in the U.S.”
“These approvals represent an important medical advance with the use of innovative cell-based gene therapies to target potentially devastating diseases and improve public health,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research.
“Today’s actions follow rigorous evaluations of the scientific and clinical data needed to support approval,” he he wrote in the statement, “reflecting the FDA’s commitment to facilitating [the] development of safe and effective treatments for conditions with severe impacts on human health.”
“CASGEVY is a first-in-class treatment that offers the potential of a one-time transformative therapy for eligible patients with sickle cell disease,” said Kewalramani.
Dr. Mikkael Sekeres, chief of the division of hematology of Sylvester Cancer Center at the University of Miami described, “The CRISPR technology [that is] being used with this treatment for sickle cell disease — a devastating inherited illness that profoundly affects survival and quality of life — is remarkable.”
“The data from the admittedly small study in support of the treatment’s approval by the FDA are impressive, and the therapy was thought safe by a panel of independent experts,” Sekeres added.
However, the high cost and eligibility restrictions may limit accessibility.