Benzinga | May 11, 2021 12:38PM EDT

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

* Precision BioSciences Inc (NASDAQ: 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.