On December 8th, 2023, two new gene therapies received FDA approval for the treatment of sickle-cell disease (SCD), a blood disorder affecting approximately 100,000 people in the United State alone. These "sickle" shaped red blood cells restrict blood flow and limit oxygen delivery to the body's tissue, causing severe pain and organ damage.
The two therapies, Casgevy and Lyfgenia, are both created the using the patient's own stem cells, which are genetically modified and given back as a one-time infusion to the patient. The patients' must first undergo high dose chemotherapy to remove cells from the bone marrow so they may then be replaced with the modified cells. This gene modification therapy will hopefully be more accessable than the current standard of sickle cell treatment, a bone marrow transplant, which carries inscreased risks and only about 20% of patients are able to find an eligible donor. The dawn of this new gene-editing era in medicine may be monumental step forward in the future of modern healthcare.
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