'Not Mo Yang Jeokhyeolgu Byeong' Treatment Path Opened... US FDA First Approves Gene Editing Therapy

Treatment Costs Reach Tens of Billions Won, Only a Few Expected to Benefit
100,000 US Patients with 'Gyeomsang Jeokhyeolgubyung'

A treatment for sickle cell disease using gene-editing technology has been approved for the first time in the United States. The sickle cell disease treatment involves removing the relevant gene from stem cells taken from the patient so that the patient's hemoglobin can return to normal hemoglobin.

According to the New York Times (NYT) and others, the U.S. Food and Drug Administration (FDA) has approved the use of 'Casgevy,' a CRISPR-based treatment jointly developed by biotechnology companies Vertex Pharmaceuticals and CRISPR Therapeutics, for severe sickle cell disease patients aged 12 and older.

Gene scissors.
Photo by Yonhap News

Casgevy is a treatment using the Nobel Prize-winning gene-editing technology CRISPR, marking the first approval of a CRISPR-based therapy.

Sickle cell disease is one of the genetic diseases affecting Black people, caused by a mutation in the hemoglobin gene that changes the shape of red blood cells into a sickle or crescent shape. These altered red blood cells obstruct blood flow, causing pain, stroke, and organ failure.

There are approximately 100,000 patients with sickle cell disease in the United States alone, about 20% of whom have severe symptoms. It is known that 1 in 365 Black babies is born with this disease, and including Black patients in Africa, millions worldwide suffer from this condition.

Casgevy treats the disease by removing the relevant gene from stem cells taken from the patient so that the patient's hemoglobin can revert to the normal fetal hemoglobin.

Gene therapy is much more effective than existing treatments such as medication, blood transfusions, or bone marrow transplants. However, due to the high cost running into millions of dollars (tens of billions of Korean won), only a few are expected to benefit.

The newly approved gene-editing therapy is priced at $2.2 million (approximately 2.9 billion KRW), while conventional gene therapy costs $3.1 million (approximately 4.1 billion KRW).

To receive this expensive treatment, approval from insurance companies is required, and currently, there are very few medical institutions capable of treating sickle cell disease using gene-editing technology. Only nine medical institutions in the U.S. offer this treatment. Moreover, due to the extensive medical resources required, each institution is expected to treat only 5 to 10 patients per year.

Meanwhile, on the same day, the FDA also approved Bluebird Bio's 'Lyfgenia,' a conventional gene therapy. Lyfgenia works by injecting patients with modified gene copies to suppress the formation of defective cells and enhance the function of specific hemoglobin.

This content was produced with the assistance of AI translation services.