SMaRT™ Technology Products

VIRxSYS’s core business is the design and construction of proprietary pre-trans-splicing molecules (PTMs) that selectively trans-splice into a defined pre-messenger RNA target, rewriting the RNA to confer new function. VIRxSYS intends to partner with pharmaceutical and biotechnology companies for the commercial use of these PTMs for RNA therapeutics and molecular imaging.

RNA Therapeutics: SMaRT™ RNA therapy uses PTMs to repair mutant mRNAs from genes associated with human disease. Because its coding sequence can consist of one or more exons, a single PTM can be used to correct all the mutations in the region of the target RNA that is being replaced. PTMs can be used to deliver coding sequences with improved function and to reduce or eliminate the deleterious effects of dominant negative mutations.

VIRxSYS is currently working to develop RNA therapies for the blood coagulation disorder Hemophilia A (Factor VIII deficiency) and for Dyslipidemia.

SMaRT™ Image PTM: VIRxSYS is developing PTMs for imaging expression of a partner’s gene of interest, which can be used for any in vitro or in vivo imaging application.