ARCUS, a next-generation genome editing platform from Precision BioSciences, is a one-step gene-editing process that is simpler than CRISPR.
The first gene-editing platforms to move into the clinic include CRISPR/Cas9, TALEN and zinc finger nuclease technology — and now there is a fourth approach that is not far behind. ARC nuclease, or ARCUS, gene-editing technology was developed by Precision BioSciences. The company recently filed an Investigational New Drug (IND) application for a gene-edited allogeneic (off the shelf) CAR-T cell program targeting CD19, for which the company will be launching a phase I/IIa clinical trial in patients with acute lymphoblastic leukemia and non-Hodgkin’s lymphoma.
Precision BioSciences also recently filed an initial public offering (IPO) with a goal of raising $100 million. The company believes it can raise that much given the nature of the ARCUS technology, which it claims is an improved approach to gene editing compared with other available platforms. Specifically, the company says that its one-step engineering process is simpler, more effective and will translate to a less-expensive, mass-production approach.
The ARC nuclease synthetic enzyme, according to Precision BioSciences, is similar to a homing endonuclease but has been improved to serve as the starting point for the genome-editing process. The company says that it is small and has customizable specificity that is “incomparable.”
This funding round is the second for Precision BioSciences, which previously raised $110 million in the summer of 2018 from a long list of investors, including venture capital firms and pharma companies, such as Gilead and Amgen. Gilead also signed a separate $445 million collaboration agreement with Precision in the fall of 2018 focused on hepatitis B.