US approves world’s first gene editing therapy

 US approves world's first gene editing therapy: what disease does it treat and how does it work |  Science

The United States Food and Drug Administration (FDA) approved on Friday, December 8, the first gene editing treatment with CRISPR in the world, an innovation that will increase the life span of many people.

Therapy was called Casgevy and is the first in the country to be approved to use CRISPR technology, also known as genetic scissors for its ability to edit a patient’s genetics to treat inherited diseases.

This type of treatment, which earned its inventors the Nobel Prize in Medicine in 2020, is possible by cutting specific regions of DNA and editing mutations that cause diseases.

Casgevy: what disease does it treat?

Casgevy, developed in companies Crispr Therapeutics y Vertex Pharmaceuticalscreated to treat sickle cell anemiaa rare, inherited blood disorder that occurs when the body produces defective hemoglobins.

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In this disease, the red blood cells, which carry hemoglobin, do not change and stick together, causing them to get stuck in the blood vessels and prevent normal blood flow. Sickle cell anemia causes patients debilitating pain and, in most cases, premature death.

Alternatives against this disease are few today. Among them are bone marrow transplanta difficult intervention that requires a genetically compatible donor, such as a brother or sister.

How does therapy work?

Casgevy therapy, which is not a drug, changes the bone marrow cells of the patient with the disease so that they produce the same hemoglobin that is produced in fetuses and children.

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It is used only once, but there is no data on the exact duration of its effects. This detail will be resolved by following up the first beneficiaries in the next 15 years.

In the United States, about 100,000 people have this disease, which tends to be more prevalent in the African-American population and, to a lesser extent, in Latin Americans.

“Gene therapy promises to provide more specific and effective treatments, especially for people with rare diseases where current treatment options are limited,” said FDA official Nicole Verdun.