What you’ll learn in this white paper

Nucleic acid therapeutics, including siRNAs and antisense oligonucleotides (ASOs), have the potential to treat a wide range of diseases. However, delivering these molecules to target tissues is challenging due to their susceptibility to degradation, high molecular weight, and anionic backbone.

This white paper explores chemical modification strategies that improve the distribution and cellular uptake of nucleic acid therapeutics, including:

• GalNAc conjugation for targeted delivery to hepatocytes in the liver.
• Long-chain fatty acids such as lauric (C12), palmitic (C16), stearic (C18), and docosanoic (C22) acids to enhance extrahepatic tissue delivery.
• Cholesterol conjugation to improve cellular uptake and support tissue distribution.
• Tocopherol (vitamin E) conjugation to facilitate delivery to the liver and, when administered appropriately, to the brain.

Why download:

• Learn how GalNAc conjugation enables precise liver targeting for siRNAs and ASOs.
• Explore lipophilic modifiers like long-chain fatty acids, cholesterol, and tocopherol that enhance cellular uptake and delivery to extrahepatic tissues.
• Understand the strategies researchers are using to expand the therapeutic reach of nucleic acid drugs.

Download the full white paper today.