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: 

     ●      Specific Binding to Liver Cells:

GalNAc specifically binds to the asialoglycoprotein receptors (ASGPR) that are highly expressed on hepatocytes (liver cells). This binding facilitates the selective uptake of nucleic acid drugs by liver cells, ensuring targeted delivery. 

     ●      Increased Cellular Uptake:
By attaching GalNAc to small nucleic acids (such as siRNA or ASOs), the drug is more efficiently internalized into liver cells via receptor-mediated endocytosis. This enhances the bioavailability of the drug in the liver. 

     ●      Improved Pharmacokinetics:
GalNAc conjugation increases the stability of the drug in circulation, allowing it to remain in the bloodstream longer before being cleared. This extended half-life improves the therapeutic window, increasing the drug's effectiveness. 

     ●      Reduced Off-Target Effects:
The liver-specific targeting of GalNAc reduces the likelihood of the drug affecting other tissues, minimizing off-target effects and systemic toxicity. 

     ●      Enhanced Efficacy:
The precise delivery to liver cells allows for higher local concentrations of the drug, leading to more efficient gene silencing (for RNAi drugs) or modulation (for ASOs) in liver-related diseases. 

By directing the therapeutic nucleic acid to the liver specifically, GalNAc enables small nucleic acid drugs to achieve higher therapeutic efficacy while minimizing side effects, particularly for diseases affecting the liver. 

GenCefe Biotech provides high-quality siRNA, miRNA, sgRNA, and custom RNA synthesis services. We can design and synthesize RNA Oligos of different lengths, different forms, and with various modifications according to customer’s needs, and also synthesize RNA sequences designed by customers. The RNA products we deliver are all purified by HPLC. ISO 9001-certified facilities and comprehensive quality control reports ensure the delivery of high-quality products.