For thousands of years, humans have been plagued by a highly dangerous blood disorder called Sickle Cell Anemia. However, there is hope on the horizon, as two new gene therapies have been discovered to cure it. Sickle cell anemia is a recessive blood disorder where healthy blood cells become stiff and sickleshaped, causing blood clots that can deprive tissue of oxygen, leading to infections and organ damage. 

Casgevy is the first of two treatments for sickle cell anemia. It resolves sickle cell anemia by taking a stem cell from the patient and rewrites the DNA to create healthy blood cells. First, the doctors extract a stem cell from the patient. Then in a lab, they edit the cell to produce healthy blood cells. Before they can transfer the healthy stem cell back into the patient doctors, use a weaker version of chemotherapy to reset the stem cells. That crucial step allows the healthy stem cell to travel back to the bone marrow from the IV injected into the blood stream. Once settled into the bone marrow, it will start producing healthy blood cells, and over time, it will out compete the sickle cells as they die. Fetal hemoglobin is immune to becoming sickle cells. The reason is because sickle cell anemia is a mutation that occurs in the β-globin gene while Fetal hemoglobin does not have that gene. Instead, it has γ-globin chains not β-globin chains. While Casgevy uses gene editing, Lyfgenia cures the disorder in another way.

Lyfgenia is the second of the two treatments for sickle cell anemia. It cures the patient from the disorder by using a virus that adds a working hemoglobin gene. This gene adds to the preexisting gene and makes the stem cells only produce anti-sickling hemoglobin. The process for this is done by first collecting a stem cell or blood cell. Next,  the doctors infect the cell with a modified virus that adds the anti-sickling hemoglobin gene. From there,  they test the cell to make sure it functions properly. After that, a controlled dose of chemotherapy is used to remove the sickle cell from making stem cells. Finally, this allows the gene to be injected into the blood stream and travel to the bone marrow. From there, it will produce anti-sickling hemoglobin blood cells.

Sickle cell anemia once was a fatal disorder but now with Lyfgenia and Casgevy gene therapies it is now curable. Due to the process of editing and adding genes, patients are now able to look forward to their future without the symptoms of sickle cell anemia looming over them.

For more information go to: lyfgenia.com or casgevy.com