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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: SMaRTTM 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.
SMaRTTM 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.
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