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Anastasia Khvorova


UMassMed Faculty Page


Title: Branched Oligonucleotides as Therapeutics. UMMS16-45; Patent Pending. 

  • This technology is based on the discovery that branched oligonucleotide structure improves the level of tissue retention in brain by more than 100 fold compared to non-branched compounds of identical chemical composition. The invention discloses branched oligonucleotides, specifically di-branched assymetric fully modified siRNAs, exhibiting great uniform distribution throughout the CNS and other target tissues, enhanced cellular uptake, minimal immune response and off-target effects, without formulation. 

Title: Novel Metabolically Stable Oligonucleotide Conjugates. UMMS16-09; Patent Pending. 

  • The invention discloses novel hydrophobically-conjugated oligonucleotides useful for RNA interference. The oligonucleotide conjugates are characterized by efficient RISC entry, minimum immune response and off-target effects, efficient cellular uptake without formulation, and efficient and specific tissue distribution. 

Title: Developing of Soluble FLT1 Targeting Therapeutics for Treatment of Preeclampsia. UMMS15-50. Patent Pending.

  • Selective elimination (filtration) of circulation "soluble fms-like tyrosine kinase 1" proteins (sFLT1s) in women with preeclampsia is a successful strategy for treatment of preeclampsia. The invention comprises oligonucleotide compounds that bind intronic regions of mRNA encoding sFLT1 protein, thereby inhibiting expression of sFLT1 protein for treatment of preeclampsia and other angiogenic disorders. Targeting these regions with RNAi compounds enables selective silencing of truncated, soluble variants without interfering with regular FLT1 function. Further, there is no detectable transfer of the oligonucleotides to the fetus. 

Title: Fully Stabilized Assimetric siRNA Compounds: an Optimal Scaffold for Conjugate Mediated Delivery. UMMS15-25; Patent Pending. 

  • This invention discloses hydrophobically-modified siRNA, featuring an advanced stabilization pattern, "hsiRNA-ASP". These siRNA compounds having the following properties: (1) fully chemically stabilized (i.e., no unmodified 2'-OH residues); (2) asymmetry; (3) 11-16 base pair duplexes; (4) alternating patten of chemicically-modified nucleotides (e.g., 2'-flour and 2'-methoxy modifications); (5) single-stranded, fully phosphorothioated tails of 5-8 bases. siRNAs with these structural properties show a dramatic enhancement in potency (5-10) fold in unassisted delivery. Also embodied are hsiRNA-ASPs conjugated to targeting agents including, but not limited to, cholesterol. Further, alteration of hsiRNA-ASP PS content has a major impact on tissue distribution and oligonucleotide uptake making these compounds very attractive therapeutic agents. 


  • This new invention spans from the discovery of potent silencing site that was previously unidentified on Huntington gene (htt) through screening of more than 200 compounds. Conventional siRNAs will target this 3’ UTR site for potent inhibition.  Efficacy was demonstrated using different siRNA configurations in several cell types and primary neurons that may aid research and future therapeutic applications. 

Title: Efficient Exosomal Loading Using Hydrophobically Modified Therapeutic Oligonucleotides. UMMS14-09; Patent Pending.

  • This novel invention discloses a novel RNAi delivery vehicle. Specifically, describing methods of loading exosomes with hydrophobically modified nucleic acids which exhibit a much higher loading efficiency than methods currently used (i.e. electroporation, transfection with cationic lipid reagents, and ultracentrifugation). 


Innovation TopicsGene TherapyRNAi/micro-RNA Therapy, Brain diseasesNeurodegerative disorder, Gene therapy improvement, Eclampsia/PreeclampsiaObstetrics, Huntingtin’s disease