United States. the first treatment with Crispr molecular scissors is allowed

US health authorities on Friday (Dec. 8) approved two treatments for sickle cell anemia, an inherited genetic blood disorder, including the first treatment using Crispr’s molecular scissors genome-editing technique.

“Gene therapy holds the promise of providing more targeted and effective treatments, especially for people with rare diseases.Nicole Verdun, who is responsible within the framework of the American Medicines Agency (FDA), emphasized in the press release.

“Transforming Patients’ Lives”

“The potential for these products to change the lives of patients with sickle cell disease is enormous“, he added. The development of this molecular scissor won the 2020 Nobel Prize in Chemistry for Frenchman Emmanuelle Charpentier and American Jennifer Doudna.

Crispr technology has revolutionized genome manipulation due to its precision and ease of use compared to 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’s treatment was already approved by British health authorities in November.

The patient’s cells are modified using Crispr technology and then transplanted into the individual. Sickle cell disease especially affects African-Americans, the American agency emphasizes. Chronic anemia, acute painful crises, and increased risk of infections are some of the most common symptoms of sickle cell disease. The consequences, with damage to vital organs, can be serious, even fatal.