Abstract

Spherical Nucleic Acids - Activating the Immune System

David Giljohann, CEO, Exicure

Exicure develops spherical nucleic acid (SNA) constructs, which are 3-dimensional arrangements of oligonucleotides where the nucleic acids are densely packed and radially oriented around a nanoparticle. AST-008 is an SNA configuration of a toll-like receptor 9 (TLR9) agonist oligonucleotide, designed to trigger anti-tumor immune responses in patients with cancer. AST-008 is intended to be administered intratumorally in combination with checkpoint inhibitors for the treatment of solid tumors. AST-008 has potent antitumor activity as a monotherapy and synergizes with anti-PD-1 antibody therapy in several preclinical tumor models. Clinical progress with our Phase Ib trial will be discussed.