Toasting a year of breakthroughs (and a few breakdowns) in Silicon Valley and beyond.
People with the genetic disease have new opportunities to eliminate their symptoms, but the treatments come with obstacles that limit their reach.
The first treatment that relies on CRISPR is expected to receive U.S. approval next month. But it may cost millions of dollars per patient.
The decision by an advisory committee may lead to Food and Drug Administration approval of the first treatment for humans that uses the CRISPR gene-editing system.
The treatment from the company Vertex would be the first medicine to use the gene editing tool CRISPR.
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