Nucleic Acid Therapies
Exicure Therapeutics develops spherical nanoparticles for the intracellular delivery of nucleic acid based therapies such as antisense RNA, siRNA, mRNA and aptomers. These particles are useful for the treatment of various immune and genetic disorders, as well as cancers.
Spherical Nucleic Acid (SNA)- SNAs are spherical nanoparticles with densely packed nucleic acids at the surface. In some cases, the particles have a gold core to which the nucleic acid is cross linked. In other cases, the particles are coreless and nucleic acid strands are held together by phosphodiester bonds. SNAs bind to class A scavenger receptors found on the surface of most cells, which allows the particles to be endocytosed into the cell endosome. Since the cell endosome contains many immunostimulatory receptors, this nucleic acid delivery approach is particularly useful for immunomodulatory therapies. SNAs are compatible with a wide range of nucleic acid therapies including anti-sense RNA, siRNA, mRNA and aptomers. Antigen can be co-conjugated with the nucleic acid for dual immunostimulation and antigen presentation effects. The SNA platform is useful for the treatment of cancers, as well as immune and genetic disorders.
Spherical Nucleic Acids: Adding A New Dimension to Nucleic Acids and Clinical Chemistry
Mirkin, CA; Petrosko, SH. Clinical Chemistry. 2018 Jun; 64(6):971-972.
Accelerating the Translation of Nanomaterials in Biomedicine
Mitagorti, S; et al. ACS Nanotechnology. 2015 Jul 28; 9(7): 6644-54.
Therapeutic Applications of Spherical Nucleic Acids
Barnaby, SN; Sita, TL; Petrosko, SH; Stegh, AH; Mirkin, CA. Cancer Treatment Research. 2015; 166: 23-50.
Original Science Research:
Exicure, inc. is the assignee on 17 patents related to their spherical nucleic acid particles. See all 17 patents.
Clinical Trials (Phase 1): Solid Tumor (TLR9 Agonist), Psoriasis (anti-IL7RA), Dermatology (anti-TNFa)
Preclinical Development: Spinal muscular atrophy (SMN2), Ataxias/Batten Disease (multiple targets), Undisclosed ophthalmology, Undisclosed GI IBD, Undisclosed pulmonology
Exicure may be interested in industry partnerships to expand the SNA platform to further diseases and may be interested in industry or academic partnerships to help improve the SNA platform.