REDWOOD CITY, Calif., Oct. 7, 2020 /PRNewswire/ -- Synthego, the genome engineering company, today announced the design of new foundational technology for standardized precision and control of CRISPR-based gene editing inside cells using light. Described in Nature Communications, Synthego researchers developed a new class of guide molecules, CRISPRoff™, a synthetic sgRNA that fragments in response to light, enabling precise temporal and spatial control of double-strand breaks created during gene editing. The publication follows Synthego's extension of the collaboration with the National Institute of Standards and Technology (NIST) Genome Editing Consortium to further standards for precise and reproducible gene editing in therapeutic development.
"CRISPRoff and our NIST Consortium collaboration highlight our deep commitment and ability to create cutting-edge CRISPR platforms to standardize predictable gene editing across a variety of disease areas, helping drive the industry forward," said Robert Deans, Ph.D., chief scientific officer of Synthego. "The ability to control off-target vulnerability and optimize CRISPR technology is critical, especially as Synthego platforms increasingly support clinical trials."
The in vitro research published in Nature Communications demonstrated that CRISPRoff is effective across multiple genomic targets in multiple cell lines and has the potential to optimize on-target editing events and the ability to spatially pattern cells in vitro.
Unlike existing control strategies, CRISPRoff does not require additional engineering steps or changes to nucleases, enabling its fitting within existing CRISPR gene editing workflows without any modifications. CRISPRoff can also be chemically tuned to respond to different wavelengths of light, which also allows for multiplexed genome engineering. Importantly, the research also showed that the implementation of CRISPRoff enables modulation but does not impact genome editing efficiency, and it can be produced in a Good Manufacturing Practice (GMP)-grade format.
"To achieve safe and controlled genome editing for the development of therapeutics, it is imperative that rigorous standards are implemented to increase the precision and reproducibility in both research and development applications," said Samantha Maragh, the lead of NIST's programs focusing on genome editing technologies. "As NIST is working on establishing standards for genome editing technologies, our collective efforts are focused on developing reliable benchmarks for assay qualification and community norms that can increase reproducibility."
Synthego is a genome engineering company that enables the acceleration of life science research and development in the pursuit of improved human health. The company leverages machine learning, automation, and gene editing to build platforms for science at scale. With its foundations in engineering disciplines, the company's platforms vertically integrate proprietary hardware, software, bioinformatics, chemistries, and molecular biology to advance life sciences from basic research through therapeutic development programs. By providing commercial and academic researchers, and therapeutic developers with unprecedented access to cutting-edge genome engineering products and services, Synthego is at the forefront of innovation in engineered biology.