Regeneron is expanding its genetic medicine efforts and will work with California startup Mammoth Biosciences on gene-editing programs reaching parts of the human body beyond the liver, the companies announced Thursday.
While CRISPR/Cas9 gene-editing technology has won a Nobel Prize, biopharma companies have struggled to turn the tool into therapies that can reach many of the body’s tissues and organs. Instead, drug developers have crowded into disease targets in the liver, the clearinghouse organ where delivery systems like lipid nanoparticles or viral vectors typically wind up.
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