FDA APPROVES FIRST CRISPR THERAPY

Summary from the TLDR newsletter

The FDA has approved two gene therapies for treating sickle cell disease. These treatments have been shown to be highly effective at preventing vaso-occlusive events and crises. One of the treatments, the first CRISPR/Cas9 therapy approved in the US, turns on the production of fetal hemoglobin to replace deformed red blood cells. The other therapy uses a Lentiviral vector to insert the genetic code for a modified type of hemoglobin designed to be anti-sickling.