Precision BioSciences Inc (DTIL)

[MI Dendream]

Precision is leading the way to editing genes in non-human primates (macaque monkeys). Long term safety and implantation have been confirmed over 3 years with demonstrated benefit on seromarkers of efficacy.

https://www.genengnews.com/news/preclinical-study-shows-safety-efficacy-and-durability-of-lowering-ldl-cholesterol-levels-long-term-in-non-human-primates/

I-Cre-I includes a built-in safety switch that shuts it off after a single, specific DNA edit reducing the risk of off-target edits.

The research team monitored NHPs for more than three years and have continued to show a sustained reduction in LDL-c levels and stable gene editing without any obvious adverse effects. After the single treatment, NHPs continue to show reductions of up to 85% in PCSK9 protein levels and a 56% in LDL-c 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,” said Derek Jantz, PhD, co-author on the paper and chief scientific officer at Precision BioSciences.

“ARCUS has attributes that we believe significantly differentiate it from RNAi or conventional AAV gene therapy approaches, as well as CRISPR gene editing approaches. At more than three years out, we are seeing a stable gene edit that is being inherited by subsequent generations of hepatocytes, and evidence thus far supports that this is a permanent change. We look forward to continued monitoring of these animals and applying these learnings to our other in vivo gene editing programs,” says Jantz.