UK regulators have authorized CRISPR Therapeutics’ Casgevy to treat two debilitating blood disorders — making it the world’s first approved CRISPR therapy.
“I hope this represents the first of many applications of this Nobel Prize winning technology to benefit eligible patients with serious diseases,” said Samarth Kulkarni, CEO of CRISPR Therapeutics.
The targets: Your cells need oxygen to function, and it’s the job of hemoglobin, a protein found in your red blood cells, to deliver it.
Sickle cell disease (SCD) and beta thalassemia (BT) are genetic disorders disrupting hemoglobin production. In SCD, the body produces abnormal hemoglobin that causes red blood cells to be rigid, sticky, and curved, or sickle shaped, instead of round — these cells aren’t as effective at carrying oxygen. In BT, the body doesn’t produce enough hemoglobin, period.
With both diseases, the body doesn’t get as much oxygen as it needs — this can lead to fatigue and weakness. With SCD, blood cells can build up and get stuck in tiny blood vessels, causing episodes of severe pain and swelling. In severe cases, patients can experience organ damage, infections, and blindness.