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FDA Approves Two Cell-Based SCD Gene Therapies

January 17, 2024

February 2024

The U.S. Food and Drug Administration (FDA) approved exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel) for the treatment of sickle cell disease (SCD) in patients 12 years or older. They are the first cell-based gene therapies to receive approval as SCD treatments.

Exa-cel is a cell-based gene therapy that modifies hematopoietic stem cells (HSCs) via genome editing using CRISPR/Cas9 technology. This is the first therapy using CRISPR/Cas9 technology to be approved by the FDA.

Approved in the treatment of patients with SCD with recurrent vaso-occlusive crises, exa-cel increases the production of fetal hemoglobin and prevents the sickling of red blood cells. After the HSCs are modified, they are engrafted within the bone marrow.

Lovo-cel is another cell-based gene therapy that uses a lentiviral vector for genetic modification. With this treatment, HSCs are modified to produce HbAT87Q, which works similarly to hemoglobin A. Lovo-cel reduces the risk of sickling and occluded blood flow. Much like exa-cel, this treatment is indicated for patients with SCD and a history of vaso-occlusive crises.

For both exa-cel and lovo-cel, patients will receive myeloablative conditioning post-HSC collection before treatment.

“These approvals represent an important medical advance with the use of innovative cell-based gene therapies to target potentially devastating diseases and improve public health,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research.

Source: FDA, December 8, 2023.

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