FDA Approves Gene-Editing Therapies for Sickle Cell Disease

by Karen Faulkner, Worthy News Correspondent

(Worthy News) – The US Food and Drug Administration (FDA) announced last week that it has approved Casgevy and Lyfgenia, two groundbreaking, gene-editing treatments for sickle cell disease (SCD) in patients 12 years and older. Sickle cell disease is a group of inherited blood disorders which affects around 100,000 people in the US.

In a Dec. 8 press release, the FDA said Casgevy represents an “innovative advancement” in the field of gene therapy. “Casgevy is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of genome editing technology,” the press release reads. “Patients’ hematopoietic (blood) stem cells are modified by genome editing using CRISPR/Cas9 technology.” Casgevy will be used for patients suffering recurrent vaso-occlusive crises.

Lyfgenia is a cell-based gene therapy, the FDA press release explains. “Lyfgenia uses a lentiviral vector (gene delivery vehicle) for genetic modification and is approved for the treatment of patients 12 years of age and older with sickle cell disease and a history of vaso-occlusive events,” the FDA says.

Both Casgevy and Lyfgenia products are made from the patients’ own blood stem cells, the FDA said. The stem cells are modified and given back as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant.

Explaining that the FDA’s approval of the two therapies follows “rigorous evaluations of the scientific and clinical data,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, is quoted in the press release as saying: “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.”

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