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Exicure Breakthrough

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The ‘Koosh ball’ that can cure.
David Giljohann has developed a breakthrough that has the ability to infiltrate a sick cell to correct the genes inside and make it healthy—or kill it.

DNA-altering drugs have been a fascination in medicine since the human genome was decoded, but until now these medications have been hitting an impenetrable wall. “The challenge was no one could figure out how to effectively deliver DNA into the cell,” explains David Giljohann. His company, Skokie-based Exicure, has over 150 patents on a technology that is solving the cellular-level delivery challenge.

His product is a “spherical nucleic acid,” or “SNA construct” for short. It’s a configuration of DNA arranged around a nanoparticle, which the 37-year-old CEO says “looks like a Koosh ball.” This unique 3D design has the ability to infiltrate a sick cell to correct the genes inside and make it healthy—or kill it. Giljohann discovered the phenomenon in 2006 with his Northwestern University graduate school classmates in the lab of Chad Mirkin, an industry leader in science and entrepreneurship who is also a co-founder of Exicure. “It’s a major, major discovery,” says Mirkin. “One that will really change the course of pharmaceutical development.”

Four drugs are being tested in patients in the U.S., the U.K. and Germany, including a clinical trial at Northwestern for brain cancer. Other trials focus on general oncology and the topical treatment of psoriasis and atopic dermatitis. Giljohann hopes the technology will revolutionize the standard of care for any genetically based disease, including cancer. “If you look at chemotherapeutics, your goal is to kill the cancer cells faster than you kill every other cell in the body. The idea behind Exicure technologies for the SNA, as well as the DNA space in general, is let’s just kill the bad cells.”

When his medicines start coming to market in the mid-2020s, Giljohann anticipates he’ll be pioneering treatments for illnesses that currently have none and introducing drugs that are more targeted, easier to administer, safer and less expensive. “From a business standpoint, that’s nirvana,” Mirkin says.

Exicure has only 30 team members but operates at the pace of a company of 300. Thanks to the human genome codes, the team is able to digitally design, test and prototype a medicine before building it. Instead of making a thousand designs for testing, they make only one, shortening the development cycle and driving down costs.
An adjunct professor in chemical and biological engineering at Northwestern, Giljohann lives in Evanston, “although our running joke at the company is that I live on seat 7A on American Airlines.” He racked up 128 flights to and from O’Hare International Airport last year visiting clinical trial sites, giving scientific talks and visiting his impressive list of investors, including Bill Gates, Eric Lefkofsky, Pat Ryan and David Walt. Exicure has raised over $100 million and became a publicly traded company in May.  Click here for a link to the original article in Crain’s Chicago Business

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