Precision BioSciences is focused on applying the proprietary ARCUS genome editing technology to cure genetic and infectious diseases where no adequate treatments exist. Gene editing technology has the incredible power to permanently cure devastating diseases; however, because gene edits are permanent, the risk of off-target or off-tissue gene edits can present a significant safety challenge.
Unlike some gene editing technologies that were designed primarily as research tools and subsequently engineered for therapeutic use, ARCUS was designed with therapeutic safety, delivery and control in mind.
Precision BioSciences has developed a robust pipeline of gene correction therapy candidates with a focus on diseases that have a clearly established genetic cause.
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Therapeutic-grade genome editing
In contrast to editing platforms that focus on ease of design and quick turnaround, the ARCUS approach is centered on safety, delivery, and control of edits – the things that we believe matter most to patients and their physicians.
ARCUS nucleases have the potential to deliver safer, more specific genetic edits by leveraging the properties of a naturally occurring gene editing enzyme – the homing endonuclease I-CreI – which evolved in nature to make a single, highly specific DNA edit before using its built-in safety switch to shut itself off.
CRISPR: What is the future of gene editing?
CRISPR allows scientists to perform surgery on DNA. For people with genetic diseases it has transformed lives with corrections that aren’t passed on to others. But should the technology be used in ways that permanently transform the human race?
While it’s probably most famous for its role in gene editing, CRISPR does more than just that: its ability to precisely cut and alter DNA could lead to new antibiotics, faster diagnosis tools, and more.