The United States Food and Drug Administration (FDA) has granted approval for a gene-editing treatment designed for sickle cell patients aged 12 and above.

The announcement, made through an FDA press release on Saturday, introduces two stages of treatment named Casgevy and Lyfgenia.

These represent the pioneering cell-based gene therapies for sickle cell disease in patients aged 12 and older.

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Casgevy, one of the therapies, stands out as the first FDA-approved treatment employing a novel genome editing technology, marking a notable advancement in the realm of gene therapy.

Nicole Verdun, the director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research, emphasized the significance of these therapies, stating, “Sickle cell disease is a rare, debilitating, and life-threatening blood disorder with significant unmet need, and we are excited to advance the field.”

Verdun highlighted the potential of gene therapy in delivering more targeted and effective treatments, especially for individuals with rare diseases where current options are limited.

Casgevy, a cell-based gene therapy, has received approval for treating sickle cell disease in patients aged 12 and older experiencing recurrent vaso-occlusive crises.

This therapy is distinctive for being the first FDA-approved treatment utilizing CRISPR/Cas9, a genome editing technology.

Explaining the process further, Verdun detailed that patients’ hematopoietic stem cells undergo modification through genome editing using CRISPR/Cas9 technology.

This technology allows precise editing of DNA, including removal, addition, or replacement of genetic material.

The modified blood stem cells are then transplanted back into the patient, where they attach and multiply within the bone marrow, increasing the production of fetal hemoglobin (HbF).

Elevated levels of HbF help prevent the sickling of red blood cells in patients with sickle cell disease.

Lyfgenia, another cell-based gene therapy, uses a lentiviral vector for genetic modification and is approved for treating sickle cell disease in patients aged 12 and older with a history of vaso-occlusive events.

This therapy involves genetic modification of the patient’s blood stem cells to produce HbAT87Q, a gene-therapy-derived hemoglobin that functions similarly to hemoglobin A found in individuals not affected by sickle cell disease.

Both Casgevy and Lyfgenia are derived from the patient’s own blood stem cells, modified and given back as a one-time, single-dose infusion as part of a hematopoietic stem cell transplant.

Before treatment, the patient’s own stem cells are collected, and a myeloablative conditioning process, involving high-dose chemotherapy, is performed to remove cells from the bone marrow.

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These cells are then replaced with the modified cells in Casgevy and Lyfgenia.

Patients who undergo Casgevy or Lyfgenia treatment will be closely monitored in a long-term study to assess the safety and effectiveness of each product.

Sickle cell disease, a group of inherited blood disorders, affects around 100,000 people in the U.S., with a higher prevalence among African Americans and, to a lesser extent, Hispanic Americans.