FDA Approves Gene Editing Treatment for Sickle Cell Disease

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The Food and Drug Administration approved two gene therapy treatments for sickle cell disease.

For Context: Sickle cell is an inherited blood disease that causes organ failure, anemia, strokes and excruciating pain. The disease affects an estimated 100,000 Americans, most of whom are black, according to Reuters (Center bias).

Details: One of the approved treatments, called Exa-cel, is a gene editing treatment. This is the first gene editing treatment ever used on humans, according to the New York Times (Lean Left bias). Outlets across the spectrum are emphasizing the scientific achievement and potential impact on those suffering from sickle cell, while also noting the barriers likely to prevent many from accessing the treatment. The New York Times noted the “onerous” procedures involved in the treatment and the “multimillion-dollar” price tag. The New York Post (Lean Right bias) noted potential side effects from the treatments, such as infertility.

Key Quotes: The New York Times quoted Dr. Stephan Grupp, chief of the cellular therapy and transplant section at Children’s Hospital of Philadelphia, stating, “It is practically a miracle that this is even possible.”

How the Media Covered It: The New York Times heavily covered the intense treatment process and high cost but did not note the potential side effects included in coverage from Reuters and the New York Post.

Why the Difference? Left-rated outlets more often highlight the financial burden of healthcare, while right-rated outlets more often highlight the potential dangers of newly developed medical treatments.

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On Friday, the Food and Drug Administration approved the first gene editing therapy ever to be used in humans, for sickle cell disease, a debilitating blood disorder caused by a single mutated gene.

The agency also approved a second treatment using conventional gene therapy for sickle cell that does not use gene editing.

For the 100,000 Americans with the disease, most of them Black, the approvals offer hope for finally living without an affliction that causes excruciating pain, organ damage and strokes.

The U.S. Food and Drug Administration (FDA) on Friday approved two gene therapies for sickle cell disease, making one of them the first treatment in the United States based on the Nobel Prize-winning CRISPR gene editing technology.

Casgevy, developed by partners Vertex Pharmaceuticals (VRTX.O) and CRISPR Therapeutics (CRSP.BN), and bluebird bio's (BLUE.O) Lyfgenia were approved for people aged 12 years and older.

For the first time in history, there’s hope that the debilitating illness known as sickle cell disease may be treatable — even cured.

The Food and Drug Administration announced Friday that two gene therapies for sickle cell disease have been approved following years of clinical trials.

Sickle cell disease is an inherited blood disorder caused by a mutated gene that affects hemoglobin, a protein in red blood cells, according to Johns Hopkins Medicine.