US health authorities have approved two treatments for sickle cell disease, an inherited genetic blood disorder, including the first treatment to use CRISPR, a genome-editing technique.
"These treatments represent a significant advance in the field of gene therapy for patients with sickle cell disease, a rare and devastating blood disorder that affects about 100,000 people in the United States," Peter Marks, head of the US Food and Drug Administration, said at a news conference. drugs (FDA).
"The potential for these products to change the lives of patients suffering from sickle cell disease is enormous," he added.
The development of this technology earned Frenchman Emmanuelle Charpentier and American Jennifer Doudna the 2020 Nobel Prize in Chemistry.
CRISPR technology has revolutionized genome manipulation thanks to its precision and greater ease of use than previous tools.
Both treatments, Casgevy and Lyfgenia, have been evaluated in clinical trials and are FDA-approved for patients 12 years of age and older.
Casgevy treatment was already approved in November by the UK health authorities.
The patient's cells are modified using CRISPR technology and then transplanted back into the patient.
As the US agency points out, sickle cell anemia mostly affects African Americans.
The most common symptoms of sickle cell disease include chronic anemia, acute pain attacks, and an increased risk of infections. The consequences of damage to vital organs can be serious, even fatal. /BGNES
