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Precision Bio's ARCUS Gene Editing Platform Is Safe, Durable; Long-Term Animal Study Shows

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Vandana Singh
·1 min read
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  • Precision BioSciences Inc (NASDAQ: DTIL) has announced three-year follow-up data showing long-term stable reduction of low-density lipoprotein (LDL) cholesterol levels in nonhuman primates (NHPs) after in vivo gene editing of the PCSK9 gene with its ARCUS platform. The company published the data online in Molecular Therapy.

  • NHPs have demonstrated a continuous and sustained reduction in LDL cholesterol levels while maintaining stable gene editing without any noticeable adverse effects. After the one-time vector administration more than three years ago, NHPs treated with ARCUS have experienced stable reductions of up to 85% in PCSK9 protein levels and a 56% reduction of LDL cholesterol levels.

  • "To our knowledge, this is the longest duration gene editing data in a large animal model. The data demonstrates that a single administration of an ARCUS nuclease could represent a potential one-time, permanent treatment for familial hypercholesteremia," commented Derek Jantz, Ph.D., co-author of the paper and Chief Scientific Officer at Precision BioSciences.

  • ARCUS is a proprietary genome editing that uses sequence-specific DNA-cutting enzymes, or nucleases, designed to either insert (knock-in), remove (knock-out), or repair the DNA of living cell organisms.

  • Last year in November, the company announced a license agreement with Eli Lilly And Co (NYSE: LLY) for the ARCUS platform for Duchenne muscular dystrophy and two other undisclosed gene targets.

  • Price Action: DTIL shares are trading higher by 7.68% at $12.20 in the premarket session on the last check Friday.

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