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U.S. poised to approve first gene-editing treatment in breakthrough for sickle cell patients - TL;DR CNBC

U.S. poised to approve first gene-editing treatment in breakthrough for sickle cell patients

Publishing timestamp: 2023-12-07 09:14:26


Summary

The U.S. Food and Drug Administration is expected to approve the exa-cel gene-editing treatment for sickle cell disease, which would be the first approved medicine in the U.S. to use CRISPR gene-editing technology. The treatment, co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics, aims to alleviate the symptoms of sickle cell disease by editing a person's DNA. The approval of exa-cel could be a breakthrough for patients and a scientific milestone, but it also presents a major test for the American healthcare system due to its high cost of around $2 million per patient.


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Keywords: businesssciencebluebird bio incbiotech and pharmaceuticalscrispr therapeutics agbusiness newsunited stateshealth care industryvertex pharmaceuticals inc

Source: https://www.cnbc.com/2023/12/07/crispr-gene-editing-treatment-us-approval.html


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