US FDA approves use of world’s first gene-edited treatment

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Sickle cell disease gene editing treatment ‘Kasgevi’ approved
Treatment technique using CRISPR, winner of the Nobel Prize in Chemistry
Hope to escape genetic disease… Cost of billions is an obstacle

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The U.S. Food and Drug Administration (FDA) approved the first gene editing treatment for humans on the 8th (local time).

For those suffering from genetic diseases, a new opportunity to escape the disease has opened up, but some analysts say that actual demand will not be high due to limited facilities and enormous treatment costs.

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According to the New York Times, the U.S. Food and Drug Administration on this day approved the use of Casgevy, the first gene-editing treatment for sickle cell disease, a debilitating blood disease caused by a single mutated gene.

Sickle cell disease is a genetic disease that causes various diseases by reducing the number of red blood cells. It is said that about 100,000 people suffer from it in the United States alone, and the majority of those suffering from the disease are black.

NYT reported that they finally have hope that they can live free from diseases that cause extreme pain, organ damage, and stroke.

Kasgebi is a treatment jointly developed by Vertex Pharmaceuticals of the United States and CRISPR Therapeutics of Switzerland, and edits genes (DNA) using CRISPR, a gene editing technology that won the Nobel Prize in Chemistry.

In clinical trials, it was reported to be effective in preventing or correcting actual mutations.

However, for actual patients, there are many obstacles to overcome. The number of medical institutions that can use this treatment is extremely limited, and the treatment process is not easy because genes must be edited or added to each patient.

Moreover, the cost can reach millions of dollars. Vertex said that the cost of gene editing treatment is around $2.2 to $3.1 million (about 2.9 to 4 billion won).

In the end, experts say that only a small number of people will actually be able to use the new treatment. The FDA believes this treatment may be available to affected patients ages 12 and older.

Meanwhile, the FDA also approved the use of Lyfgenia, a sickle cell disease treatment using existing gene therapy.


Source: Donga

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