The Food and Drug Administration has approved the world’s first medicine employing CRISPR gene-editing technology to treat sickle cell disease, giving thousands of Americans new hope to treat the painful disease.

“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” said Nicole Verdun, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research.

BIDEN ASSERTS AUTHORITY TO CLAIM PATENTS OF DRUGS DEVELOPED WITH FEDERAL RESEARCH

Two new treatments, Casgevy and Lyfgenia, were approved on Friday in a long-anticipated move that tackles genetic disorders by turning off or replacing specific genes.

Approximately 100,000 Americans are affected by sickle cell disease, which is the result of an inherited genetic mutation that produced a dysfunctional form of the protein hemoglobin, which carries oxygen in the bloodstream. Hemoglobin proteins in those affected with the disease is shaped in the form of a sickle, giving the disorder its name.

Casgevy targets the specific gene that causes the dysfunction, switching it off and allowing for the production of fetal hemoglobin proteins. Lyfgenia works by producing a gene-therapy derived hemoglobin that functions similarly to the adult form of the protein.

Both products come from a patient’s blood stem cells, which are delivered in a one-time, single-dose infusion once they have been genetically modified.

“Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited,” said Verdun.

Sickle cell disease occurs most frequently in black populations, affecting about one in every 365 black or African American births in the United States. The disorder also occurs in one in every 16,300 Hispanic-American births, according to the Centers for Disease Control and Prevention.

CLICK HERE TO READ MORE FROM THE WASHINGTON EXAMINER

Sickle cell disease occurs mostly among descendants from parts of the world where malaria is endemic. Genetic carriers for sickle cell disease, also referred to as those with the sickle cell trait, have some protective advantages against malaria.

“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, director of the FDA’s Center for Biologics Evaluation and Research.

Original Location: FDA approves world’s first CRISPR gene-editing drug for sickle-cell disease

Washington Examiner Videos