FDA treatment using CRISPR molecular scissor

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Illustration representing the CRISPR molecular scissors in action.

Agence France-Presse

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American health authorities have approved two treatments to treat sickle cell anemia, a genetic blood disease, including the first associated with the molecular scissor CRISPR, a genome editing technique.

These treatments represent a major breakthrough in gene therapy for patients with sickle cell disease, a rare and debilitating blood disease that affects about 100,000 people in the United States, Peter said at a news conference. Marks, head of the United States Medicines Agency (FDA).

The potential for these products to transform the lives of patients living with sickle cell disease is enormous.

A quote from Peter Marks, head of the US Medicines Agency

The development of this molecular scissor earned the Nobel Prize in Chemistry to the Frenchwoman Emmanuelle Charpentier and the American Jennifer Doudna in 2020.

CRISPR technology has revolutionized genome manipulation through its precision and greater ease of use compared to previous tools.

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The patient's cells are modified using CRISPR technology, then transplanted again into the individual.

Both treatments, Casgevy and Lyfgenia, have been evaluated in clinical trials and are approved by the FDA for patients ages 12 and up. years.

The Casgevy treatment had already been approved in November by the British health authorities.

Sickle cell disease particularly affects African-Americans, underlines the American agency.

Chronic anemia, acute painful crises and increased risk Infections are among the most common symptoms of sickle cell disease. The repercussions, with damage to vital organs, can be serious, even fatal.