OUR APPROACH | ARCUS Genome Editing

What is ARCUS?


ARCUS is a next-generation genome editing platform derived from a natural genome editing enzyme called a homing endonuclease.

Homing endonucleases

Homing endonucleases are nature’s genome editing system. They are site-specific DNA-cutting enzymes encoded in the genomes of many eukaryotic species. Homing endonucleases have the unusual ability to precisely recognize long DNA sequences (12-40 base pairs) that are typically rare enough to occur only once in a complex genome. These non-destructive enzymes trigger gene conversion events that modify the genome in a very precise way, most frequently by the insertion of a new DNA sequence. The ability to target a single DNA break in a complex genome and to achieve gene modification without random off-targeting makes homing endonucleases the ideal starting material for a therapeutic-grade genome editing technology.

ARCUS

The backbone of the ARCUS technology is the ARC nuclease – a fully synthetic enzyme similar to a homing endonuclease but significantly improved to be the starting point for the first therapeutic-grade genome editing platform. The ARC nuclease is small, has unparalleled specificity, and can be customized to recognize a DNA sequence within any target gene. ARC nucleases are created using a set of proprietary in silico and lab-based techniques to ensure maximum gene editing efficiency with minimum off-target activity. Importantly, ARC nucleases can be optimized to control potency and specificity based on the analysis of cutting activity in a relevant model organism.

What makes ARCUS unique in the world of genome editing?


Proprietary

Precision BioSciences owns ARCUS. Only a Precisioneer can make an ARCUS reagent.

Multi-functional

ARCUS isn’t limited to deletions. ARCUS can be used to insert, delete or edit DNA.

Specific

ARCUS doesn’t miss its target. ARCUS doesn’t generate truly random DNA-breaks.

Small

310 amino acids. 930 base pairs. ARCUS is compatible with essentially any delivery strategy.

One

Polypeptide. ARCUS is a single protein expressed from a single gene. There’s no need to deliver multiple parts.

Seamless

Matched 4 base pair 3′ overhangs result in perfect, predictable removal of DNA.