Precision BioSciences Shares Move Higher As ARCUS Platform Shows Promising Gene Editing Approach For Transthyretin Amyloidosis

Precision BioSciences Inc DTIL has announced new in vivo data using ARCUS genome editing platform resulting from a preclinical research collaboration with investigators at the University of Pennsylvania.
Data were presented at the 2021 American Society of Genetic & Cell Therapy (ASGCT) Annual Meeting.
Researchers reported preclinical results using ARCUS to knock out the transthyretin (TTR) gene responsible for transthyretin amyloidosis (ATTR), a rare disease that leads to peripheral neuropathy or cardiomyopathy.
In ARCUS-treated no-human primates (NHPs), high levels of genomic editing were achieved, resulting in substantial and persistent serum TTR reductions.
Up to 46% indels were found at the genomic DNA locus, translating to ~80% editing of the TTR messenger RNA transcripts maintained between liver biopsies collected at 18 and 128 days post-AAV administration.
Researchers found that high editing efficacy of the TTR gene was reflected in serum TTR levels, where there was an associated reduction of greater than 95% by day 21 post-vector administration in three out of the four ARCUS-treated NHPs.
These serum TTR reductions endured to 250 days after a single AAV administration.
Price Action: DTIL shares are up 11.5% at $9 during the market trading session on the last check Tuesday.