Precision Bio’s ARCUS gene editing platform is safe and durable; Long-term animal study exhibitions
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Precision BioSciences Inc (NASDAQ: DTIL) announced Follow-up dates for three years shows a long-term stable lowering of the low-density lipoprotein (LDL) cholesterol level in non-human primates (NHPs) after in vivo gene editing of the PCSK9 gene with its ARCUS platform. The company published the data online in Molecular Therapy.
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NHPs have shown continuous and sustained lowering of LDL cholesterol levels while maintaining stable gene editing with no noticeable side effects. After single vector administration more than three years ago, NHPs treated with ARCUS experienced stable reductions in PCSK9 protein levels of up to 85% and reductions in LDL cholesterol levels of 56%.
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“To the best of our knowledge, these are the dates with the longest gene editing duration in a large animal model. The data show that a single administration of an ARCUS nuclease could be a potential one-time, long-term treatment for familial hypercholesterolemia, ”commented Derek Jantz, Ph .D., Article co-author and chief scientific officer at Precision BioSciences.
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ARCUS is proprietary genome editing that uses sequence-specific DNA-cutting enzymes or nucleases designed to either knock-in, knock-out, or repair DNA from living cell organisms.
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Last 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 unknown gene targets.
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Price action: DTIL shares are trading 7.68% at $ 12.20 in the premarket session on the final Check Friday.
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