A research collaboration between the university’s Center for Genetic Diseases and the Chicagoland biotech company Exicure Inc. aims to discover successful new therapeutics for genetic diseases.

The center, led by Michelle Hastings, PhD, an expert in RNA biology and regulation of gene expression, is working to understand the mechanisms of dysfunction in inherited disorders and to discover treatments using small molecule and nucleic acid-based approaches. It will work closely with Exicure, which is developing a new class of immunomodulatory and gene-silencing drugs against validated targets.

It is estimated that more than 10,000 rare diseases that strike millions of people across the globe are caused by an error in a single gene in the human DNA, according to the World Health Organization. Many of these disorders have no effective treatment.

“Tomorrow’s treatments begin with today’s research,” said Â鶹ӰÒô Executive Vice President for Research Ronald Kaplan, PhD. “We are strongly encouraged by the novel research underway in our Center for Genetic Diseases. Our collaboration with Exicure builds on both groups’ expertise in the field of oligonucleotides as a solution to these often deadly diseases.”

Dr. Hastings has developed patented antisense oligonucleotide technologies for disorders such as Usher syndrome, which causes hearing and vision loss, and the devastating childhood disorders, Batten disease and cystic fibrosis. Antisense technology is based on a therapeutic platform that has potential applications to a large number of human conditions, several of which are under investigation in the Hastings lab.

“New genetic diseases are being discovered at a rapid rate,” Dr. Hastings said. “Our goal is to deliver treatment options for people diagnosed with or at risk for a genetic disease.”

The initial partnership will focus on genetic diseases using Exicure’s spherical nucleic acid (SNATM) architecture, designed to unlock the potential of therapeutic oligonucleotides in a wide range of cells and tissues.

Exicure CEO David Giljohann, PhD, said, “We’re fortunate to be working with a world leader in genetic medicines, so close to our research hub in Chicago, to drive new pathways to innovation and the identification of successful new therapeutics.”