GENE THERAPY & RNAi-BASED THERAPY

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. In practice, a non-functional/mutated gene is replaced with an engineered DNA molecule encoding a functional copy or a therapeutic protein. RNA interference (RNAi)-based therapy uses engineered RNA molecules to inhibit in vivo gene expression. In practice, the expression or injection of engineered RNA molecules work via mechanisms including transcript cleavage, sequestration, and inhibition of protein translation. Impactys provides a wide range of gene & RNAi-based therapeutic solutions, including:

  • Engineered RNA Molecules
    • Small-interfering RNAs (siRNAs)
    • Short-hairpin RNAs (shRNAs)
    • microRNA agomiRs & antagomiRs
    • Double-stranded RNAs (dsRNAs)
    • Antisense RNAs (asRNAs)
  • Chemical Modifications
    • 2ʹ sugar modifications (2ʹ-O-methyl, 2ʹ-fluoro (2ʹ-F), 2ʹ-O-(2-methoxyethyl)(2ʹ-MOE))
    • Backbone modifications (phosphorothioate linkages)
  • Delivery Technologies
    • Nanoparticles, lipid nanoparticles (LNPs)
    • Polymers
    • Dendrimers
    • Nucleic acid nanostructures
    • Exosomes
    • Conjugated melittin-like peptides (MLPs)
    • Targeting ligands (aptamers, antibodies, peptides, and small molecules)