The U.S. Food and Drug Administration (FDA) has recently given its approval for two groundbreaking treatments, Lyfgenia and Casgevy, offering new hope to patients with sickle cell disease.
Lyfgenia and Casgevy represent a significant leap in treating sickle cell disease, a hereditary blood disorder affecting around 100,000 Americans. Lyfgenia, developed by Bluebird Bio, employs gene-editing techniques to eliminate pain crises associated with the condition. Casgevy, a collaboration between Vertex Pharmaceuticals and CRISPR Therapeutics, uses CRISPR technology to turn on fetal hemoglobin, allowing red blood cells to maintain a healthy shape and alleviate pain crises in most patients.
The FDA has encouraged post-approval studies by both Bluebird Bio and Vertex Pharmaceuticals to monitor patients for 15 years, emphasizing the importance of understanding the therapies’ long-term impact and potential side effects.
Source : https://www.researchandmarkets.com/issues/fda-approves-breakthrough-gene-therapies-for
