FDA approves Casgevy and Lyfgenia for sickle cell disease
The FDA approved Casgevy and Lyfgenia for patients 12 and older with sickle cell disease, the first cell-based gene therapies cleared for the condition. Sickle cell disease affects about 100,000 people in the United States. The disorder causes red blood cells to take on a crescent shape that restricts blood flow and oxygen delivery to tissues. Casgevy is the first FDA-approved treatment built on CRISPR/Cas9 genome editing technology, targeting the recurrent vaso-occlusive crises that mark the disease.
Patients with severe sickle cell disease now have two approved gene therapy options in the United States.
Source: FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease (fda.gov).
Written by the Genomes desk from the primary source linked above and checked against it. Research use only; not medical advice. Corrections: [email protected].
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