Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today
The U.S. Food and Drug Administration has just given its approval for two groundbreaking treatments, Casgevy and Lyfgenia, which mark a significant milestone in the field of gene therapy. These treatments are specifically designed to combat sickle cell disease (SCD) in patients aged 12 and above. What makes Casgevy even more remarkable is that it is the first-ever FDA-approved treatment to utilize a cutting-edge genome editing technology, showcasing a remarkable leap forward in the realm of gene therapy.
“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” said Nicole Verdun, M.D., director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research. “Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”
Sickle cell disease is a collection of inherited blood disorders that affect around 100,000 individuals in the United States. While it is most prevalent among African Americans, it also impacts Hispanic Americans, albeit to a lesser extent. The primary issue in sickle cell disease lies in a mutation within hemoglobin, a vital protein found in red blood cells responsible for delivering oxygen to the body's tissues.
This mutation causes the red blood cells to adopt a crescent or "sickle" shape. Consequently, these sickled red blood cells impede blood flow within the vessels, resulting in limited oxygen supply to the body's tissues. This, in turn, leads to excruciating pain and organ damage known as vaso-occlusive events (VOEs) or vaso-occlusive crises (VOCs). The recurrence of these events can pose life-threatening risks and potentially result in severe disabilities or premature death.
Casgevy, a revolutionary cell-based gene therapy, has been granted approval for the treatment of sickle cell disease in patients aged 12 and above who experience recurrent vaso-occlusive crises.
What sets Casgevy apart is that it is the first-ever therapy approved by the FDA that utilizes CRISPR/Cas9, a groundbreaking genome editing technology. By employing CRISPR/Cas9, scientists can precisely target specific areas of DNA and make accurate modifications such as removal, addition, or replacement.
The modified blood stem cells are then reintroduced into the patient's body, where they attach and multiply within the bone marrow. This process leads to an increase in the production of fetal hemoglobin (HbF), a special type of hemoglobin that aids in the delivery of oxygen. In individuals with sickle cell disease, elevated levels of HbF effectively prevent the sickling of red blood cells.
Another remarkable cell-based gene therapy called Lyfgenia also exists. Lyfgenia employs a lentiviral vector, which acts as a gene delivery vehicle for genetic modification. It has received approval for treating patients aged 12 and above who have sickle cell disease and a history of vaso-occlusive events.
Both Casgevy and Lyfgenia are developed using the patient's own blood stem cells. These cells undergo modification and are then administered back to the patient as a one-time, single-dose infusion during a hematopoietic (blood) stem cell transplant. If you're interested in learning more about these groundbreaking therapies, you can visit this link.
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