{"id":125128,"date":"2023-12-08T12:05:04","date_gmt":"2023-12-08T17:05:04","guid":{"rendered":"http:\/\/kendallharmon.net\/?p=125128"},"modified":"2023-12-09T07:57:15","modified_gmt":"2023-12-09T12:57:15","slug":"stat-in-historic-decision-fda-approves-a-crispr-based-medicine-for-treatment-of-sickle-cell-disease","status":"publish","type":"post","link":"https:\/\/kendallharmon.net\/?p=125128","title":{"rendered":"(Stat) In historic decision, FDA approves a CRISPR-based medicine for treatment of sickle cell disease"},"content":{"rendered":"

The Food and Drug Administration on Friday approved the world\u2019s first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for\u00a0sickle cell disease<\/a>\u00a0that delivers a potential cure for people born with the chronic and life-shortening blood disorder.<\/p>\n

The\u00a0new medicine<\/a>, called Casgevy, is made by Vertex Pharmaceuticals and CRISPR Therapeutics. Its authorization is a\u00a0scientific triumph<\/a>\u00a0for the technology that can efficiently and precisely repair DNA mutations \u2014 ushering in a new era of genetic medicines for inherited diseases.<\/p>\n

In a clinical trial, Casgevy was shown to eliminate recurrent episodes of debilitating pain caused by sickle cell, which afflicts approximately 100,000 people in the U.S., a vast majority of whom are Black. The therapy, whose scientific name is exa-cel, is described as a potential cure because the genetic fix enabled by CRISPR is designed to last a lifetime, although confirmation will require years of follow-up.<\/p>\n

Read it all<\/a>.<\/p>\n

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The FDA has approved the world\u2019s first medicine based on #CRISPR<\/a> gene-editing technology, a groundbreaking treatment for sickle cell disease. 🧵 https:\/\/t.co\/01YGdXybX8<\/a><\/p>\n

— STAT (@statnews) December 8, 2023<\/a><\/p><\/blockquote>\n