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The US Food and Drug Administration approved two gene-based treatments for sickle cell disease Friday, including the first therapy that uses the gene-editing technique CRISPR,...
Using CRISPR, it edits the DNA found in a patient’s stem cells to remove the gene that causes the disease. “The patient is their own donor,” Thompson said. “It’s a game-changer,” said ...
The Vertex-CRISPR therapy has a U.S. list price of $2.2 million, while bluebird's is $3.1 million. Both therapies, pitched as one-time treatments, will be available in early 2024.
Early research in animal models suggest that therapies based on CRISPR technology have potential to treat a wide range of diseases, including cancer, progeria, beta-thalassemia, sickle cell disease, hemophilia, cystic fibrosis, Duchenne's muscular dystrophy, Huntington's disease, transthyretin amyloidosis and heart disease.
EDIT-101 is a CRISPR based gene therapy for treatment of Leber congenital amaurosis, which is currently in clinical trials. EDIT-301 is an experimental potential treatment utilizing the firm's CAS 12a editing technology for sickle cell disease and beta-thalassemia .
This allows DNA to be specifically modified and edited, which can be used to ameliorate diseases. CRISPR Therapeutics is applying this technology platform to research, develop and commercialize medicines for various diseases including sickle cell disease, beta thalassemia, various cancers, type 1 diabetes, and cardiovascular diseases.
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