Caribou Biosciences of Berkeley, CA, is developing a technology based on the bacterial nuclease Cas9, which generates double-stranded DNA breaks in a sequence-specific fashion.
Caribou cofounder and CEO Rachel Haurwitz, Ph.D. says, “The efficiency and the ease of use of the Cas9-sgRNA system has allowed rapid and exciting work across a number of cell types and organisms already this year, but questions around specificity have lingered,” she commented.
“It is clear that at least for some implementations of the technology, improvements on specificity will be necessary, and the double-nicking strategy is an exciting example of such an improvement.”
Caribou founders are all from UC Berkeley, and include James Berger, Ph.D. (now at Johns Hopkins), Dr. Doudna, Martin Jinek, Ph.D. (now at the University of Zurich), and Dr. Haurwitz.
Dr. Haurwitz told GEN, “There is broad applicability for CRISPR technology; we have seen a flurry of activity in the academic and the commercial spaces for reagents and kits that enable site-specific engineering.” Further, she predicts, there will be increasing demands for modified animals that can serve as human disease models. “We have already partnered with SAGE Labs so that SAGE can offer researchers an expanded portfolio of animal models and services generated using the Cas9 technology.”
Beyond the immediate term research uses, Dr. Haurwitz says, “We see broad applicability across strain engineering of microbes, agricultural biotechnology, and human therapeutics, specifically gene therapy.”
She says that Caribou is “focusing on broadening and deepening the platform so it can be applicable to these spaces.” It’s clear that getting a handle on off-target effects, that is, being able to measure them and understand what they mean and what their implications are for a given system are critical. She says, “The needs will be difficult for human therapeutic developers. Understanding the tolerance or range of acceptability for off-target effects and then developing the tools to measure them really needs to be done.”
Last September, SAGE Labs, a provider of in vivo products, services and technologies in the field of genome engineering, said it had entered into a partnership and licensing agreement with Caribou for key intellectual property related to the CRISPR/Cas9 genome editing system.
The agreement gave SAGE Labs exclusive rights to Caribou's intellectual property to produce and sell genetically engineered rats, and nonexclusive rights for mouse and rabbit models. The Cas9 system, the company says, will complement SAGE's ZFN technology, allowing SAGE to offer customers access to novel animal models of human disease.
Meanwhile, we expect that activity in the CRISPR space will continue to increase as further refinements to the technology accrue.