FDA approves first cell-based gene therapies for sickle cell disease
For the first time, the FDA has approved two cell-based gene therapies for treating the rare but potentially fatal sickle cell disease. One is also the first approved therapy to utilize the CRISPR/Cas9 gene editing technology, signifying innovative advancement in the fields of gene therapy and regenerative medicine.Sickle cell disease (SCD) is a group of inherited blood disorders that affect hemoglobin, the oxygen-carrying protein in red blood cells. In someone with SCD, the hemoglobin is mutated, causing the red blood…