Suggestions

Portfolio covering most prevalent types of therapeutic oligonucleotides

  • Antisense oligonucleotides (ASOs)
    • siRNA 
    • miRNA
    • Gapmers/RNA-DNA Chimera
    • Aptamers
  • Oligonucleotide-peptide/lipid/sugar conjugates for tissue specific API delivery
  • sgRNA for CRISPR/Cas technology
  • Immunostimulant ONs/CpG oligonucleotides (ISOs)

Modifications covering

  • Sugar modifications, i. e. LNA, 2´-F, 2´-OMe, 2´MOE, …
  • Backbone modifications, i. e. Thiolation (PTO) …
  • 5´and internal modifications, i. e. 5mC, 5-hydroxyC, 8-bromoG,
  • 8-aminoC, 2-thioT, GalNac, Cholesterol, PEG, Peptide, …
  • 3´ modifications, i. e. GalNac, Cholesterol, PEG, Peptide, …



Figure 1. N-acetylgalactosamine molecule (in gray) conjugated ON to increase delivery to the hepatocytes of the liver (1).




Figure 2. Lipophilic-siRNA conjugate for delivery to extrahepatic tissues (2).



Figure 3. Peptide oligonucleotide conjugate (POC, 3).

References

  1. Ryan Cross. Have antisense oligonucleotides hit their stride? Chemical & Engineering News. 2018. https://cen.acs.org/business/antisense-oligonucleotides-hit-stride/96/i45 © Ionis Pharmaceuticals and 2024 American Chemical Society.
  2. Vasant Jadhav and Martin Maier. Behind the Paper - Expanding the Reach of RNAi Therapeutics with Next Generation Lipophilic siRNA Conjugates. Research Communities by Springer Nature. 2024. https://communities.springernature.com/posts/expanding-the-reach-of-rnai-therapeutics-with-next-generation-lipophilic-sirna-conjugate.
  3. MacCulloch T., Buchberger A., Stephanopoulos N.. Emerging applications of peptide-oligonucleotide conjugates: bioactive scaffolds, self-assembling systems, and hybrid nanomaterials. Org Biomol Chem. 2019 Feb 13;17(7):1668-1682.