General structure of a nucleoside, involving multiple GalNAc moieties with acetyl groups (OAc, AcO) and other functional groups connected through linkages (Z, Y, X )
What is GalNAc? GalNAc (N-Acetylgalactosamine) is a monosaccharide derivative that serves as a high-affinity targeting ligand for the asialoglycoprotein receptor (ASGPR). This receptor is predominantly and selectively expressed on the surface of hepatocytes (liver cells). By conjugating therapeutic agents to GalNAc, these molecules gain the ability to be efficiently and specifically internalized into liver cells, making GalNAc a cornerstone technology in modern targeted drug delivery.
Synthesis and Conjugation
Our GalNAc products are engineered through a sophisticated and reproducible process:
Chemical Synthesis: GalNAc ligands are synthesized using advanced organic chemistry techniques to ensure high purity and precise molecular structure. This includes the preparation of mono-, di-, and tri-valent GalNAc constructs to fine-tune binding affinity to ASGPR.
Functionalization for Conjugation: The ligands are furnished with reactive terminal groups (e.g., DBCO, azide, NHS ester, maleimide), enabling facile and stable conjugation to a wide array of therapeutic payloads via click chemistry or other bioconjugation techniques.
Quality Assurance: Every batch is subjected to rigorous analytical characterization (HPLC, MS, NMR) to confirm identity, purity, and conjugation readiness, ensuring optimal performance in downstream applications.
Role in Targeted Therapeutics GalNAc is utilized to create targeted conjugates for a new class of therapeutics. It is typically covalently linked to the 3' or 5' end of oligonucleotide-based drugs (like siRNA or ASOs) or other therapeutic modalities. This conjugation is a critical step in the manufacturing process, transforming systemically administered drugs into precise liver-targeting medicines.
Our Advantages
We provide GalNAc technologies that translate directly into enhanced therapeutic potential and streamlined development for our clients:
Unmatched Targeting Specificity & Efficiency:
Our GalNAc ligands exhibit exceptionally high affinity for the ASGPR, ensuring maximal delivery of the therapeutic payload to hepatocytes. This dramatically reduces the required systemic dose, minimizing off-target effects and enhancing patient safety.
Therapeutic Enhancement
By leveraging GalNAc technology, you can significantly increase the potency and therapeutic index of your liver-targeted drugs, a benefit clearly demonstrated by multiple approved therapeutics on the market.
Comprehensive & Customizable Portfolio
We offer a diverse range of GalNAc ligands (mono to triantennary), spacers (PEG, alkyl), and conjugation handles to perfectly match your drug's chemistry and desired pharmacokinetic profile.
Scalable cGMP Manufacturing
We support your journey from discovery to clinic with scalable synthesis under rigorous cGMP conditions, providing the quality and documentation needed for therapeutic development.
Applications of Our GalNAc
Our GalNAc ligands are revolutionizing the development of targeted agents across multiple modalities:
Oligonucleotide Therapeutics
siRNA Therapeutics: Conjugation to siRNA enables robust, durable gene silencing in the liver with very low doses, as validated by approved drugs.
Antisense Oligonucleotides (ASOs): Enhances the delivery and efficacy of ASOs for knocking down pathogenic mRNAs in hepatocytes.
mRNA Therapeutics: Guides therapeutic mRNA constructs to the liver for targeted protein expression or vaccination.
Other Targeted Therapeutic Modalities
Peptide and Protein Delivery: Facilitates liver-specific delivery of peptide-based drugs, enzymes, or proteins for treating metabolic diseases.
Small Molecule Delivery: Used to target small molecule prodrugs to the liver, potentially improving the treatment of liver cancers, fibrosis, or infections.
Diagnostic & Imaging Agents
Liver-Specific Imaging Contrast Agents: GalNAc conjugation can deliver imaging tags (e.g., fluorophores, radiolabels) specifically to hepatocytes, enabling high-contrast detection of liver diseases.
Research Tools
Mechanistic Studies of Liver Biology: Used to study ASGPR function, endocytic pathways, and intracellular trafficking in liver cells.
Target Validation: Serves as a critical tool to validate liver-specific gene targets in preclinical models.
