Overview: The Gateway to Undruggable Targets

Therapeutic oligonucleotides represent a transformative class of precision medicines capable of selectively modulating gene expression to treat diseases previously considered "undruggable". The therapeutic potential of these nucleic acid polymers is immense, but their success hinges entirely on one critical factor: effective delivery. At CD BioGlyco, our therapeutic oligonucleotide delivery development service is a comprehensive, end-to-end platform designed to optimize oligonucleotide stability, enhance tissue-specific uptake, and ensure efficient endosomal escape, rapidly accelerating your candidate from discovery to preclinical success. We partner with clients to custom-design highly potent and safe delivery systems tailored to their specific therapeutic targets.

Core Technologies

Our platform employs cutting-edge delivery modalities focused on maximizing stability, specificity, and intracellular bioavailability. Conjugates are chemically defined entities where the oligonucleotide is covalently linked to a specific targeting ligand.

• N-acetylgalactosamine (GalNAc) Conjugates

This is the gold standard for robust, high-affinity hepatocyte targeting. The triantennary GalNAc ligand avidly binds to the asialoglycoprotein receptor (ASGPR), which is highly expressed on the surface of liver cells. This binding triggers highly efficient, clathrin-mediated endocytosis. CD BioGlyco focuses on designing metabolically stable GalNAc ligands with innovative anomeric linkages (e.g., S-, C-, or N-glycosides) to prevent premature cleavage by glycosidases in the endosome, thereby sustaining the therapeutic effect and enhancing in vivo efficacy.

• Peptide Conjugates

Utilizing cell-penetrating peptides (CPPs) or receptor-targeting peptides to facilitate uptake and enhance endosomal escape, particularly important for extrahepatic tissues like the central nervous system (CNS) and muscle.

• Lipid Conjugates

Short lipid chains attached to the oligonucleotide can improve lipophilicity, promoting interaction with cell membranes for uptake in tissues like the CNS, lung, and eye.

Custom Solutions Across all Delivery Modalities

Our integrated, stage-gate process ensures methodical design and robust validation for every delivery system. It begins with target and oligonucleotide design, using in silico analysis for sequence selection and chemical modifications (e.g., 2'-MOE, LNA) to optimize potency and stability. Next, delivery system engineering focuses on designing targeting ligands (e.g., GalNAc, peptides) and linkers, or formulating LNPs with specific lipids for effective biodistribution. The synthesis and formulation stage covers large-scale oligonucleotide production, conjugation/encapsulation, and rigorous purification and QC (e.g., HPLC, mass spectrometry). In vitro assessment evaluates efficacy through cellular uptake and target knockdown studies, alongside stability tests. Finally, in vivo PK/PD & safety studies assess drug exposure, pharmacological activity, and toxicology in animal models. This comprehensive approach guarantees a rationally developed and thoroughly validated therapeutic candidate. To support advanced conjugation projects, we provide custom synthesis services for novel, non-natural, and metabolically stabilized GalNAc Ligands. We offer unparalleled flexibility and depth in delivery system development, categorized by ligand type, chemical reaction, and oligonucleotide payload.

• By GalNAc Type

• By Chemical Reaction

• By Oligonucleotide Type

• Other Delivery

Workflow: A Phased Approach to Optimized Delivery

Publication Data

Applications

• Research Therapy Development

This service is pivotal for advancing investigational oligonucleotide-based therapies from concept to validation. It supports academic and industrial research programs aimed at creating new intervention strategies.

• Pharmaceutical Drug Development

It is extensively applied in the discovery and optimization of oligonucleotide drug candidates. This includes processes like lead identification, sequence optimization, and formulation to progress novel pharmaceutical agents.

• Gene Function and Regulation Studies

These services provide essential tools for functional genomics. By enabling the efficient delivery of oligonucleotides, researchers can modulate gene expression to study gene function, signaling pathways, and regulatory mechanisms in vitro and in vivo.

• Disease Mechanism Investigation

The platform is used to explore the molecular underpinnings of various pathologies. Targeting specific genes or proteins involved in a disease state facilitates the study of disease progression and identifies potential points of intervention.

• Delivery System Optimization

A core application is the dedicated research and improvement of oligonucleotide delivery technologies. This focuses on enhancing targeting specificity, cellular uptake, and stability to overcome biological barriers.

Advantages

• Many Years of Specialization

Our scientific leadership possesses decades of experience in nucleic acid chemistry and delivery, giving us a proprietary edge in rational design and problem-solving.

• Superior GalNAc Ligand Chemistry

Our focus on metabolically stable GalNAc derivatives ensures prolonged residence time and maximal therapeutic effect in vivo, offering a significant potency advantage over conventional approaches.

• Integrated PK/PD/Toxicity Assessment

Our service includes crucial preclinical validation steps—detailed biodistribution analysis, accurate PK/PD correlation, and comprehensive safety evaluations—to de-risk your program early.

• Proven Track Record

We have a history of successfully enabling the development of oligonucleotide-based research therapeutics for our clients, demonstrating our ability to solve complex delivery problems.

• Customized Approach

We recognize that each oligonucleotide candidate is unique. Our strategies are tailored to the specific properties of your molecule and its intended biological target.

Frequently Asked Questions

Customer Review

• "The triantennary GalNAc conjugate developed by the CD BioGlyco team was truly groundbreaking. It achieved a 90% knockdown in our target gene at a dose far lower than what we had seen with previous collaborators. Their metabolic stability data gave us the confidence to move quickly to in vivo studies."

— Dr. Schmidt, Senior Scientist, Pharmacology Department

• "We engaged CD BioGlyco for a complex extrahepatic LNP delivery challenge. Their ability to rapidly screen novel ionizable lipids and optimize the formulation provided a clear path forward for our CNS-targeted mRNA program. Exceptional technical depth."

— Manager, R&D Strategy Division

• "The purification and QC of the final GalNAc-ASO conjugate were flawless. The batch-to-batch consistency was the best we've experienced, ensuring our toxicology studies are based on a reliable, high-purity product."

— Director, Process Chemistry Unit

Associated Services

Our therapeutic oligonucleotide delivery development service focuses on optimizing the transport and cellular uptake of nucleic acid therapeutics, the efficacy of which is fundamentally dependent on their intrinsic chemical properties. To enable precise customization of these molecular attributes, we provide a specialized Oligonucleotide-based Bases Modification Service featuring:

• Affinity-plus Base Modification for enhanced target binding specificity
• Inosine Modification to modulate hybridization characteristics
• 2'-MOE and 2'-O-Methyl RNA base modification for improved nuclease resistance
• Fluoro Bases Modification to fine-tune pharmacokinetic profiles

This integrated approach ensures the development of oligonucleotides with optimized stability, activity, and delivery efficiency.

At CD BioGlyco, our delivery development service is built on a foundation of proprietary chemistry, robust engineering, and two decades of biological expertise. Whether your goal is highly efficient liver targeting via metabolically stable GalNAc conjugates or pioneering delivery to extrahepatic tissues using cutting-edge LNPs and peptides, contact us. We provide the integrated platform and scientific partnership required to translate your nucleic acid therapeutic from a potent sequence into a viable drug candidate.