Lakhasly

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."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.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.Much like exa-cel, this treatment is indicated for patients with SCD and a history of vaso-occlusive crises.

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.