New CRISPR-based sickle cell treatment, explained

The Food and Drug Administration has approved the world’s first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease that delivers a potential cure for people born with the chronic and life-shortening blood disorder.

The new medicine, called Casgevy, is made by Vertex Pharmaceuticals and CRISPR Therapeutics. Its authorization is a scientific triumph for the technology that can efficiently and precisely repair DNA mutations — ushering in a new era of genetic medicines for inherited diseases.

This new video from STAT explains how the drug works, and how it is manufactured.

More STAT coverage on this treatment here: https://www.statnews.com/2023/12/08/f...

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Founded in 2015, STAT is a global digital media brand that focuses on delivering fast, deep, and tough-minded journalism about the life sciences industries to over six million monthly site visitors and an additional 20 million readers on the Apple News app. STAT takes you inside academic labs, biotech boardrooms, and political backrooms, casting a critical eye on scientific discoveries, scrutinizing corporate strategies, and chronicling the roiling battles for talent, money, and market share. With an award-winning newsroom, STAT provides indispensable insights and exclusive stories on the technologies, personalities, power brokers, and political forces driving massive changes in the life sciences industry — and a revolution in human health.