Yes, several RNA-based therapeutics using GalNAc conjugation have been approved for clinical use, particularly for liver-targeted therapies. Some of the approved drugs that utilize GalNAc for targeted delivery include:
GalNAc plays a crucial role in enhancing the targeted delivery of small nucleic acid drugs, particularly for liver-targeted therapies. Here's how it works:
Various modifications are applied to small nucleic acids to enhance their stability, reduce immunogenicity, and improve targeting. Below are the most commonly used modifications and their primary functions:
Several delivery strategies are employed to overcome the challenges of nucleic acid stability, cellular uptake, and tissue targeting for small nucleic acid drugs.