SFDA Grants Approval for First Gene Therapy for Sickle Cell Anemia and Thalassemia
The Saudi Food and Drug Authority (SFDA) has approved Casgevy, a groundbreaking gene therapy for patients 12 and older with sickle cell anemia and thalassemia.
Casgevy, employing CRISPR/Cas9 genome editing, alters patients' hematopoietic stem cells to properly produce hemoglobin. The stem cells are collected, edited in a lab, and then reinserted into the patient for long-lasting results.
Sickle cell anemia, a genetic disorder, deforms red blood cells, causing pain and impaired oxygen delivery. Thalassemia, another genetic condition, disrupts hemoglobin production, leading to anemia and breathlessness. Casgevy aims to address the underlying genetic defects of these disorders.
Sickle cell anemia, a genetic disorder, deforms red blood cells, causing pain and impaired oxygen delivery. Thalassemia, another genetic condition, disrupts hemoglobin production, leading to anemia and breathlessness. Casgevy aims to address the underlying genetic defects of these disorders.
