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Major human CRISPR trial derailed by FDA—at least for now

| | June 5, 2018

You’ve heard it before: CRISPR has the potential to change our lives for the better. But first, we have to give it a chance.

In December, gene-editing company CRISPR Therapeutics announced a partnership with biotech company Vertex to develop CTX001, the world’s first gene-based treatment for sickle cell disease (SCD). Now, the U.S. Food and Drug Administration (FDA) has denied the companies’ request to move forward with a early-phase trial of CTX001 in adult volunteers, placing a “clinical hold” on the application.

According to a CRISPR Therapeutics press release, the FDA has “certain questions” it wants resolved before it gives the go-ahead to the human CRISPR study…

SCD is a genetic blood disorder in which a protein in red blood cells called hemoglobin doesn’t transport oxygen throughout the body the way it should. This can cause a host of health problems, from chronic pain to blindness to strokes.

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This trial, should it move forward, won’t be the first time CRISPR has been tested in humans — that first try was in China in 2016. But this trial would mark one of the first to happen in the U.S., as MIT Tech Review reports (several others, including some to treat SCD, are also moving forward, and it’s not clear which group will get there first).

Read full, original post: The FDA Puts the Brakes on A Major CRISPR Trial in Humans

The GLP aggregated and excerpted this article to reflect the diversity of news, opinion, and analysis. Click the link above to read the full, original article.
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