Food and Drug AdministrationFDAThe US announced this Friday the approval of two treatments for sickle cell disease (SCD), one of which is the first commercially available treatment in the country based on gene editing technology.

In a statement, the FDA announced the approval of Casgevy and Lyfgenia for counteract the effects of this genetic disease this causes the red blood cells to become stiff and sticky as they change shape from a disk to a crescent shape.

Kasgevy is also the first FDA-approved drug to uses a new type of genome editing technology“indicating innovative progress in the field of gene therapy,” the agency said.

Sickle cell anemia This is a group of hereditary blood diseases. which affects approximately 100 thousand people in the United States. It is more common among African Americans and, although less common, it also affects Hispanic Americans.

The main problem with sickle cell anemia is hemoglobin mutationa protein found in red blood cells that supplies the body’s tissues with oxygen.

sickle red blood cells restrict blood flow in blood vessels and limit the supply of oxygen to body tissues, causing severe pain and organ damage, which can lead to life-threatening disability or premature death.

The two approved treatments are created from a patient’s own blood stem cells, which are modified and reimplanted.

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For American President Joe Biden, the announcement made this Friday “is extremely promising for developing treatments “complementary treatments that save lives and give hope to millions of Americans living with other rare diseases.”

As noted in a White House statement, the FDA confirmation These two methods “represent the power of medical innovation to improve the lives of Americans.”

Living with sickle cell disease often means “putting the goals and plans of daily life on the back burner,” he said. withdrawal due to chronic pain and associated serious health complications.”

Casgevy is the first FDA-approved therapy using CRISPR/Cas9. a type of genome editing technology which can be used to cut DNA in specific areas, allowing DNA to be precisely edited (removed, added, or replaced).

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The safety and effectiveness of Kasgevy were assessed in a study involving 44 patients taking They had a serious history and the primary efficacy outcome was freedom from serious events for at least 12 consecutive months during the 24-month follow-up period.

According to Nicole Verdun, director of the Division of Therapeutic Products at the FDA’s Center for Biologics Evaluation and Research, gene therapy “promises to offer more specific and effective treatments, especially for people with rare diseases for which current treatment options are limited.” (EFE)