FDA Document Raises Concerns Over a New Gene Editing Therapy

The therapy, called exa-cel, is a new technology that would precisely edit a patient’s DNA. But in recent documents, the U.S. Food and Drug Administration has sounded the alarm about potential unintended edits that could have far-reaching health consequences.

The FDA is now getting external experts’ opinions on whether this CRISPR technology used by Vertex, a biopharmaceutical company, and CRISPR Therapeutics, a biotechnology company, is safe long-term.