FDA Okays 1st Human Gene Editing Cure For Sickle Cell Disease

The Food and Drug Administration (FDA) has approved a new sickle cell disease treatment using CRISPR genome editing technology.

Sickle cell disease is caused by a mutation in hemoglobin, a protein in red blood cells that delivers oxygen to the body's tissues.

It's the first approved human gene editing therapy, as reported by Doug Cunningham for United Press International (UPI).

The FDA said Casgevy and Lyfgenia are the two cell-based gene therapies approved for sickle cell treatment. Sickle cell disease affects approximately 100,000 people in the US annually.

"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 the FDA's Dr. Nicole Verdun.

Casgevy is the first FDA-approved therapy using CRISPR and is used to modify hematopoietic (blood) stem cells. Lyfgenia uses a gene delivery vehicle for gene modification that alters the patient's blood stem cells to produce gene-derived hemoglobin.

When added to red blood cells, that hemoglobin lowers the risk of sickling, the FDA said.

She added that the FDA is "excited to advance the field, especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies."

Sickle cell disease is caused by a mutation in hemoglobin, a protein in red blood cells that delivers oxygen to the body's tissues.

The inherited blood disorder is most common in Black Americans but also affects Hispanics. The mutated cells have a sickle shape, as seen under microscopes. They restrict blood flow, limiting oxygen delivery in the body.

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