Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug
JUNE 28, 2021
The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . In three more patients treated at 0.1mg/kg levels fell by 52%.