CRISPR’s potential to prevent or treat disease is widely recognized. But the gene-editing technology can also be used as a research tool to probe and understand diseases. That’s the basic insight behind KSQ Therapeutics. The company uses CRISPR (clustered regularly interspaced short palindromic repeats) to alter genes across millions of cells. By observing the effect of turning on and off individual genes, KSQ can decipher their role in diseases like cancer. The company uses those insights to develop new treatments. The approach allows KSQ to evaluate the function of every gene in the human genome. The approach was developed at MIT by KSQ co-founder Tim Wang (photo), PhD ’17, in the labs of professors Eric Lander, PhD, and David Sabatini, MD, PhD. “Now we can look at every single gene, which you really couldn’t do before in a human cell system, and therefore there are new aspects of biology and disease to discover, and some of these have clinical value,” says Dr. Sabatini, who is also a KSQ co-founder. KSQ’s product pipeline includes small-molecule drugs as well as cell therapies that target genetic vulnerabilities identified from their experiments with cancer and tumor cells. KSQ believes its CRISPR-based methodology gives it a more complete understanding of disease biology than other pharmaceutical companies and thus a better chance of developing effective treatments to cancer and other complex diseases. KSQ’s scientific co-founders had been studying the function of genes for years before advances in CRISPR allowed them to precisely edit genomes about 10 years ago. They immediately recognized CRISPR’s potential to help them understand the role of genes in disease biology.
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