Great news today! The United States Food and Drug Administration (FDA) has just granted approval for an incredible treatment for sickle cell illness. Casgevy is the first medication of its kind based on CRISPR gene-editing innovation and has been authorized for usage in the United Kingdom as well.
“Gene treatment holds the pledge of providing more targeted and reliable treatments, particularly for people with uncommon illness where the existing treatment alternatives are restricted,” said Doctor Nicole Verdun, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research.
Sickle cell illness is a serious condition affecting over 100,000 Americans. The only known treatment previously was a bone marrow transplant from a donor, which comes with many risks and challenges.
Casgevy utilizes the CRISPR-Cas9 gene modifying strategy and involves modifying genes with CRISPR from a client’s stem cells drawn from bone marrow. The remarkable new treatment does not include the risk of graft versus host illness the way a conventional bone marrow transplant does, offering new hope for patients.
In addition to Casgevy, the FDA has also authorized another sickle cell treatment called Lyfgenia. You can read more about this amazing medical breakthrough here.
