ASGPR ligand-1
[CAS# 1426160-58-8]

Identification

• Classification: API >> Inhibitor drug
• Name: ASGPR ligand-1
• Synonyms: 5-[(2R,3R,4R,5R,6R)-3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxy-N-[3-[3-[3-[3-[3-[5-[(2R,3R,4R,5R,6R)-3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxypentanoylamino]propylamino]-3-oxopropoxy]-2-[[3-[3-[5-[(2R,3R,4R,5R,6R)-3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxypentanoylamino]propylamino]-3-oxopropoxy]methyl]-2-aminopropoxy]propanoylamino]propyl]pentanamide
• Molecular Formula: C₆₁H₁₁₀N₁₀O₂₇
• Molecular Weight: 1415.58
• CAS Registry Number: 1426160-58-8
• SMILES: CC(=O)N[C@@H]1[C@H]([C@H]([C@H](O[C@H]1OCCCCC(=O)NCCCNC(=O)CCOCC(COCCC(=O)NCCCNC(=O)CCCCO[C@H]2[C@@H]([C@H]([C@H]([C@H](O2)CO)O)O)NC(=O)C)(COCCC(=O)NCCCNC(=O)CCCCO[C@H]3[C@@H]([C@H]([C@H]([C@H](O3)CO)O)O)NC(=O)C)N)CO)O)O

Discovory and Applicatios

ASGPR ligand-1 is a chemical compound that has emerged as a significant tool in the field of targeted drug delivery, particularly in the treatment of liver diseases. The compound functions as a ligand for the asialoglycoprotein receptor (ASGPR), a receptor predominantly expressed on the surface of hepatocytes in the liver. This specificity makes ASGPR ligand-1 a valuable component in the design of therapies that require precise delivery of therapeutic agents to the liver.

The discovery of ASGPR ligand-1 is rooted in the study of receptor-mediated endocytosis, a process by which cells internalize molecules via specific receptors on their surface. The ASGPR, known for its role in clearing glycoproteins from the bloodstream, recognizes and binds to galactose and N-acetylgalactosamine residues on glycoproteins. ASGPR ligand-1 was developed to mimic these natural ligands, allowing it to selectively bind to the ASGPR on hepatocytes. This selective binding is crucial for targeting therapeutic agents directly to the liver, thereby enhancing the efficacy of treatments for liver-specific conditions while minimizing systemic side effects.

One of the primary applications of ASGPR ligand-1 is in the targeted delivery of nucleic acids, such as small interfering RNA (siRNA) or antisense oligonucleotides, to hepatocytes. The use of nucleic acids in therapy holds promise for treating a range of genetic and infectious diseases by modulating gene expression. However, delivering these molecules to the liver in a safe and efficient manner is challenging due to their instability in the bloodstream and the difficulty in crossing cellular membranes. ASGPR ligand-1 addresses these challenges by facilitating the targeted delivery of nucleic acids to the liver, ensuring that they reach their intended site of action and thereby improving therapeutic outcomes.

In addition to its role in nucleic acid delivery, ASGPR ligand-1 is also being explored for use in the delivery of small-molecule drugs and proteins. By conjugating these therapeutic agents to ASGPR ligand-1, researchers can achieve targeted delivery to hepatocytes, potentially reducing the dosage required and minimizing off-target effects. This approach is particularly beneficial in the treatment of liver diseases such as hepatocellular carcinoma, hepatitis, and liver fibrosis, where targeted drug delivery can enhance therapeutic efficacy and reduce toxicity.

The development of ASGPR ligand-1 has also spurred interest in its use as a diagnostic tool. By attaching imaging agents to ASGPR ligand-1, it is possible to visualize and monitor liver function and disease progression in vivo. This application is particularly valuable in the early detection of liver diseases, where timely diagnosis can significantly improve patient outcomes. Additionally, ASGPR ligand-1-based imaging agents can be used to evaluate the effectiveness of liver-targeted therapies, providing a non-invasive means of assessing treatment response.

The synthesis of ASGPR ligand-1 typically involves the chemical modification of sugars or sugar derivatives to create a structure that mimics the natural ligands of the ASGPR. This process requires precise control over the stereochemistry and functional groups of the molecule to ensure high binding affinity and specificity for the receptor. Advances in synthetic chemistry have enabled the efficient production of ASGPR ligand-1, facilitating its use in both research and clinical settings.

As the field of targeted drug delivery continues to evolve, ASGPR ligand-1 is expected to play a central role in the development of new therapies for liver diseases. Its ability to selectively target hepatocytes offers a powerful tool for enhancing the delivery and efficacy of therapeutic agents, while its potential applications in diagnostics further underscore its versatility and importance in modern medicine.