Science and Technology

Exicure's Approach

Our passion for developing the next generation of nucleic acid therapies targeting RNA to address both genetic and non-genetic neurological disorders starts with our scientific staff possessing more than 100 years of joint oligonucleotide experience in bringing drugs from concept to the clinic. Our approach leverages our combined expertise against diseases and conditions with high unmet need and well-validated targets in disease pathology.

Headquartered in Chicago, IL, Exicure’s 30,000 sq ft facility houses its discovery and development efforts and is assisted by automated, high throughput screens to test thousands of oligonucleotides to our targets of interest. From our oligonucleotide chemistry expertise, we further refine our development candidates for critical characteristics of efficacy and safety. Our refined discovery and development process has enabled Exicure to identify several promising candidates in CNS conditions quickly.

In developing the next generation of nucleic acid therapeutics, Exicure utilizes its proprietary spherical nucleic acid (SNA™) platform. SNAs are nanoscale constructs consisting of synthetic nucleic acid sequences densely radiating from a spherical core. The design of our SNAs gives rise to distinct chemical and biological properties and may provide advantages over other nucleic acid therapeutics. SNA constructs overcome one of the most difficult obstacles to nucleic acid therapeutics: safe and effective delivery into cells and tissues. SNA constructs exhibit unparalleled transfection efficiency into numerous cell and tissue types without the need of transfection agents. Additionally, the SNA architecture allows versatility with a full spectrum of nucleic acid chemistries; multi-targeting capabilities; and a simple and scalable manufacturing process. With our oligonucleotide chemistry expertise, we continually pursue enhancements of our proprietary platform to further address limitation with conventional linear oligonucleotide therapeutics.