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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.
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