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Sickle cell anemia patients can now receive treatment using the gene editing technique using CRISPR/Cas 9 after it was approved by the US Food and Drug Agency (FDA). Two approved gene therapy treatments are Casgevy and Lyfgenia. It makes America the second country to allow the DNA gene editing technique to be used in the treatment of patients after the UK which also allowed its use to treat sickle cell anemia.
Through these two new therapies, the patient's stem cells taken from the bone marrow are edited with the genes that cause the disease removed. After that the patient will receive chemotherapy treatment to destroy the patient's bone marrow. Then the stem cells that have been edited are re-injected and they will produce only healthy hemoglobin without it becoming sickle cells like before.
In a clinical trial conducted last year, it was effective in 93.5% of patients 12 months after the treatment was administered. Previously sickle cell anemia was treated through marrow transplants which were difficult as a compatible donor was required first. With the CRISPR editing technique, patients become donors to their own treatments.
At this point Casgevy and Lyfgenia treatment can only be given to patients aged 12 and over. But one drawback of this brand-new treatment is its cost, which is estimated at $2.2 million per patient.