FDA approves first treatment to use gene-editing tool CRISPR

The Food and Drug Administration (FDA) has approved Casgevy, the first gene-editing therapy ever to be used in humans for treating sickle-cell disease.

It uses the Nobel-prize-winning CRISPR gene-editing tool. The treatment collects and then genetically modifies a patient’s bone marrow stem cells that are later infused into the patient.

Prevalent among Black people, sickle cell disease is a genetic disorder in which red blood cells contort into a sickle-shaped cell disease that affects hemoglobin. It causes pain, organ damage, and early death. The disease affects around 100,000 in the US and over 20 million globally.

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The therapy takes months to complete, compromises the immune system, and costs $2.2 million per patient.

Additionally
The FDA also approved Bluebird Bio’s Lyfgenia, the second treatment for sickle-cell disease. But it comes with a black-box warning that it can cause rare blood cancer. The therapy costs $3.1 million.