As a professor at the UC Davis School of Medicine highlighted, Intellia Therapeutics released data supporting the treatment of transthyretin amyloidosis (ATTR), a disorder in which protein is deposited in organs and tissue, with the first single-dose, systemically delivered CRISPR/Cas9 therapy. “The very early clinical data… suggested that the 95% reduction in the ATTR protein that they saw in non-human primates, they’re also seeing in humans, so that’s really exciting,” they said. The US Food and Drug Administration (FDA) recently granted orphan drug designation to the treatment, NTLA-2001. As the first CRISPR therapy to edit a disease-causing gene inside the human body, the FDA said NLTA-2001 “has the potential to be the first single-dose treatment for ATTR amyloidosis”, halting and reversing its devastating complications.3https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-receives-us-fda-orphan-drug-designation
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