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
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the potential results, uses and advancement of our in vivo gene editing programs and ARCUS-based gene editing technology, including, without limitation, its attributes and effects upon transthyretin amyloidosis (ATTR). In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “believe,” “could,” “eligible,” “expect,” “should,” “plan,” “intend,” “estimate,” “target,” “mission,” “goal,” “may,” “suggest,” “will,” “would,” “should,” “could,” “target,” “potential,” “project,” “predict,” “contemplate,” “potential,” or the negative thereof and similar words and expressions.
Forward-looking statements are based on management’s current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding and requirements under our current debt instruments and effects of restrictions thereunder; risks associated with raising additional capital; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical or greenhouse studies and clinical or field trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, biotechnology and agricultural biotechnology fields; our or our collaborators’ ability to identify, develop and commercialize product candidates; pending and potential liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the
All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210511005126/en/
Interim Chief Financial Officer
Senior Director, Corporate Communications