New Preclinical Data Presented at the 2021 American Society of Genetic & Cell Therapy Annual Meeting Highlights Precision BioSciences’ ARCUS In Vivo Genome Editing
Results Show Promising Gene Editing Approach for Transthyretin Amyloidosis
In the poster, “Translation of an AAV-delivered gene editing approach for transthyretin amyloidosis from mice to nonhuman primates,” researchers report positive preclinical results using ARCUS to knock out the transthyretin (TTR) gene responsible for transthyretin amyloidosis (ATTR), a rare disease that leads to peripheral neuropathy and/or cardiomyopathy.
“While there are approved treatment approaches for ATTR, these require repeated administration, whereas a gene editing approach delivered by adeno-associated viral vectors, including the one we’re studying with the ARCUS nucleus, could provide an effective one-time treatment option,” said
In ARCUS-treated 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, which was 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, suggesting the genomic edits and subsequent protein reduction may be permanent.
“Using ARCUS to knock out the TTR gene in NHPs provided an important opportunity to evaluate the persistent effects of ARCUS for in vivo editing,” said
All abstracts for the ASGCT 2021 Meeting are available on the meeting website.
ARCUS® is a proprietary genome editing technology discovered and developed by scientists at
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