Rosalind Franklin University of Medicine and Science announced today a research collaboration with the Chicagoland biotech company Exicure Inc. to develop new therapeutics for genetic diseases.
RFU’s Center for Genetic Diseases 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 RFU Executive Vice President for Research Dr. Ronald Kaplan. “We are strongly encouraged by the novel research underway in our Center for Genetic Diseases. Our collaboration with Exicure builds on both group’s expertise in the field of oligonucleotides as a solution to these often deadly diseases.”
Center Director Dr. Michelle Hastings, an expert in RNA biology and regulation of gene expression, 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 (SNA™) architecture, designed to unlock the potential of therapeutic oligonucleotides in a wide range of cells and tissues.
Exicure CEO Dr. David Giljohann said the company is fortunate to collaborate with RFU, a leading expert in the field of genetic diseases.
“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,” Dr. Giljohann said.
About Rosalind Franklin University
Rosalind Franklin University of Medicine and Science is committed to serving humanity through the interprofessional education of health and biomedical professionals and the discovery of knowledge dedicated to improving wellness. Founded in Chicago in 1912, RFU embodies the spirit of inquiry and excellence modeled by its namesake Dr. Rosalind Franklin, whose Photo 51 was crucial to solving the structure of DNA. Recognized for its research in areas including neuroscience, brain-related diseases, inherited disorders, diabetes, obesity, and gait and balance, the university encompasses the Chicago Medical School, College of Health Professions, College of Pharmacy, School of Graduate and Postdoctoral Studies and the Dr. William M. Scholl College of Podiatric Medicine. Learn more at rosalindfranklin.edu.
About Exicure Inc.
Exicure Inc. (NASDAQ: XCUR) is a clinical-stage biotechnology company developing therapeutics for immuno-oncology, inflammatory diseases and genetic disorders based on our proprietary Spherical Nucleic Acid, or SNA technology. Exicure believes that its proprietary SNA architecture has distinct chemical and biological properties that may provide advantages over other nucleic acid therapeutics and may have therapeutic potential to target diseases not typically addressed with other nucleic acid therapeutics. Exicure’s lead program is in a Phase 1b/2 trial in patients with advanced solid tumors. The company is based outside of Chicago, IL, and in Cambridge, MA.
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