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Antibody-ASO Conjugate

Antibody-ASO Conjugate

Targeted Nucleic Acid Delivery Site-Specific Conjugation Technology Integrated Bioconjugation Platform

At BOC Sciences, we provide fully integrated Antibody-ASO Conjugate (AOC) development services, empowering pharmaceutical and biotech innovators to design and produce highly specific, next-generation nucleic acid therapeutics. By combining antibody-based targeted delivery with antisense oligonucleotide (ASO) gene modulation, our platform enables precise, tissue-selective delivery of RNA therapeutics that overcome traditional biodistribution barriers. Using advanced conjugation chemistries, proprietary linker systems, and state-of-the-art analytical platforms, we deliver end-to-end solutions-from antibody and ASO design, through conjugation process development, bioanalytical characterization, and GMP-ready manufacturing-helping clients efficiently transition from concept to clinic with maximum quality, speed, and regulatory compliance.

Why Choose Antibody-ASO Conjugates?

Traditional ASO and siRNA therapies face challenges such as poor tissue selectivity, rapid clearance, and limited cellular uptake. Antibody-ASO Conjugate technology addresses these issues by:

Enhancing Target Specificity: Directs nucleic acid payloads precisely to target cells or tissues via antibody-mediated recognition.

Improving Pharmacokinetics: Extends half-life and circulation stability through optimized linker and conjugation strategies.

Boosting Cellular Uptake: Promotes receptor-mediated endocytosis, enabling higher intracellular delivery of oligonucleotides.

Reducing Off-Target Effects: Minimizes systemic exposure and associated toxicity compared to unconjugated ASOs.

Streamlining Clinical Translation: Provides a modular platform compatible with diverse ASO chemistries and antibody formats.

By integrating immunoconjugation, oligonucleotide chemistry, and biophysical analytics, we deliver tailor-made AOC solutions that overcome key barriers in RNA therapeutics.

Our AOC Service Capabilities

We provide a complete, modular suite of services to support every phase of your Antibody-ASO Conjugate (AOC) development—from discovery to GMP manufacturing.

Antibody & Target SelectionAntibody & Target Selection
  • Identification and validation of optimal cellular targets based on tissue specificity and expression profile.
  • Generation of high-affinity monoclonal, bispecific, or recombinant antibodies tailored for AOC design.
  • Screening for binding kinetics, internalization potential, and receptor recycling efficiency.
  • Target examples include TfR1, HER2, EGFR, Integrins, and CD33, depending on the indication.
ASO Design & OptimizationASO Design & Optimization
  • Custom antisense oligonucleotide, gapmer, or siRNA sequence design with in-silico off-target prediction.
  • Incorporation of chemical modifications (2'-O-methyl, phosphorothioate, 2'-MOE, LNA) to enhance nuclease resistance and potency.
  • Optimization of charge balance and hydrophobicity for efficient conjugation and cellular delivery.
  • Synthesis and purification using high-throughput solid-phase or enzymatic methods.
Conjugation Strategy DevelopmentConjugation Strategy Development
  • Development of site-specific or controlled stochastic conjugation methods to achieve precise Oligo-to-Antibody Ratio (OAR).
  • Selection and synthesis of cleavable or non-cleavable linkers depending on desired release mechanism.
  • Implementation of diverse chemistries such as click chemistry, maleimide-thiol coupling, and enzyme-mediated transglutaminase conjugation.
  • Evaluation of conjugation efficiency, payload stability, and reproducibility under GMP-compatible conditions.
Analytical Characterization & Quality ControlAnalytical Characterization & Quality Control
  • Comprehensive physicochemical characterization including LC-MS, HIC, CE, UV spectroscopy, and hybridization assays.
  • Determination of conjugation ratio, free ASO content, and product homogeneity.
  • Assessment of antibody binding affinity, internalization activity, and ASO functional integrity post-conjugation.
  • Long-term stability and forced-degradation studies to ensure formulation robustness.
Process Development & Scale-UpProcess Development & Scale-Up
  • Establishment of scalable conjugation and purification workflows for pilot and GMP batches.
  • Process optimization for yield, reproducibility, and impurity removal (e.g., free ASO, linker residues).
  • Development of formulation and lyophilization processes to maintain stability and bioactivity.
  • Preparation of CMC documentation, process validation, and regulatory compliance support for IND/CTA submissions.
Bioanalytical & PK/PD EvaluationBioanalytical & PK/PD Evaluation
  • Development of GLP-compliant bioanalytical assays for total antibody, conjugated ASO, and free ASO quantification.
  • In-vitro potency testing using gene-silencing and reporter assays in relevant cell models.
  • In-vivo pharmacokinetics (PK) and pharmacodynamics (PD) evaluation in rodent and non-rodent species.
  • Tissue distribution and biodistribution studies using labeled AOC tracking and imaging methods.

Our AOC Service Process

At BOC Sciences, we follow a streamlined, scientifically proven process to ensure the efficient, high-quality development of Antibody-ASO Conjugates.

Project Consultation & Target Definition
Project Consultation & Target Definition

We begin by assessing your project goals, therapeutic indication, and target tissue to design a customized AOC development strategy. Our experts provide scientific and regulatory input to ensure alignment with both biological and commercial objectives.

Antibody and ASO Design
Antibody and ASO Design

Our team designs or sources high-affinity antibodies and optimizes ASO sequences for potency, stability, and minimal off-target risk. We employ computational prediction tools and modification strategies (e.g., LNA, 2'-O-methyl) to enhance delivery performance.

Conjugation Chemistry Development
Conjugation Chemistry Development

We establish the optimal site-specific conjugation or controlled stochastic approach using cleavable or non-cleavable linkers. This step defines the precise Oligo-to-Antibody Ratio (OAR) and ensures consistent conjugate quality across production batches.

Analytical Characterization & Validation
Analytical Characterization & Validation

Each conjugate is thoroughly characterized using LC-MS, CE, HIC, and hybridization assays to confirm structural integrity, purity, and bioactivity. We also validate receptor binding, internalization efficiency, and gene-silencing potency.

Process Scale-Up & GMP Transition
Process Scale-Up & GMP Transition

We develop scalable, reproducible conjugation and purification workflows. CMC documentation, stability studies, and technology transfer packages are prepared to support IND/CTA submissions.

Bioanalytical & Preclinical Support
Bioanalytical & Preclinical Support

GLP-compliant bioanalytical assays are provided for PK/PD, biodistribution, and toxicology studies. We support regulatory filings with complete analytical comparability data and preclinical reporting packages.

Why Choose Our Services?

Targeted Nucleic Acid Delivery
Targeted Nucleic Acid Delivery

Our Antibody-ASO Conjugate platform enables precise, tissue-specific delivery of oligonucleotides through antibody-mediated recognition. This significantly enhances intracellular uptake while reducing systemic exposure, making RNA therapeutics safer and more effective for challenging indications such as neuromuscular and oncology disorders.

Site-Specific Conjugation Technology
Site-Specific Conjugation Technology

Using advanced site-specific conjugation chemistry, we achieve controlled attachment of ASOs to antibodies with defined stoichiometry and stability. This ensures uniform product quality, predictable pharmacokinetics, and smooth scalability from research to GMP manufacturing.

Integrated Bioconjugation Platform
Integrated Bioconjugation Platform

Our end-to-end bioconjugation workflow unifies antibody discovery, oligonucleotide synthesis, linker development, conjugation, purification, and analytics. By housing every step under one roof, we eliminate vendor fragmentation, shorten project timelines, and maintain full traceability throughout development.

Enhanced Therapeutic Precision
Enhanced Therapeutic Precision

By leveraging antibody targeting and optimized ASO chemistry, our conjugates deliver high-specificity gene silencing in desired cells while sparing healthy tissues. This translates into improved efficacy, lower dosing requirements, and superior safety margins compared to unconjugated ASO therapies.

Application Areas of Antibody-ASO Conjugate Technology

Neuromuscular Disorders
  • Precision Delivery: AOCs use antibodies targeting Transferrin Receptor 1 (TfR1) to deliver ASOs directly to skeletal and cardiac muscle.
  • Therapeutic Focus: Effective for genetic diseases such as Duchenne Muscular Dystrophy (DMD) and Myotonic Dystrophy Type 1 (DM1).
  • Key Advantage: Overcomes traditional delivery barriers, achieving high intracellular uptake and durable gene silencing in muscle tissue.
Central Nervous System (CNS) Diseases
  • Targeted Access: AOCs can cross the blood-brain barrier (BBB) using antibodies directed at receptors like TfR1 or the insulin receptor.
  • Therapeutic Use: Enables systemic administration for conditions such as ALS, Huntington's disease, and Alzheimer's disease.
  • Clinical Value: Provides a non-invasive alternative to intrathecal injections while ensuring targeted ASO delivery to neurons and glial cells.
Oncology
  • Specific Targeting: Antibodies against HER2, EGFR, or PD-L1 direct ASOs to malignant cells with high receptor expression.
  • Mode of Action: Silences oncogenic drivers or resistance-related genes, complementing antibody-drug conjugate (ADC) therapies.
  • Therapeutic Impact: Enhances tumor selectivity, reduces systemic toxicity, and opens new avenues in precision oncology.
Liver and Metabolic Disorders
  • Focused Delivery: AOCs target specific hepatocyte receptors to fine-tune ASO distribution within liver tissue.
  • Application Range: Supports treatment of NASH, familial hypercholesterolemia, and other metabolic or fibrotic diseases.
  • Key Benefit: Achieves controlled gene silencing in desired liver subtypes, improving safety and efficacy of oligonucleotide drugs.
Inflammatory and Autoimmune Diseases
  • Immune Targeting: Utilizes antibodies against CD4, CD19, or ICAM-1 to deliver ASOs to specific immune cell populations.
  • Therapeutic Use: Designed for chronic inflammatory conditions like rheumatoid arthritis, multiple sclerosis, and SLE.
  • Clinical Edge: Enables localized suppression of inflammatory mediators while preserving systemic immune balance.
Research and Diagnostic Applications
  • Versatile Platform: Provides customizable AOC constructs for cell-specific RNA delivery and silencing experiments.
  • Research Value: Ideal for mechanistic studies, target validation, and biomarker discovery.
  • Innovation Driver: Empowers scientists with a controllable system to explore RNA function and therapeutic targeting across multiple cell types.

Frequently Asked Questions (FAQ)

What is an Antibody-ASO Conjugate (AOC)?

An AOC is a hybrid molecule that links an antibody (for tissue-specific targeting) with an antisense oligonucleotide (ASO) (for gene expression regulation). This design enables highly selective delivery of RNA therapeutics to specific cells or tissues.

How is an AOC different from an Antibody-Drug Conjugate (ADC)?

Both rely on antibody targeting, but ADC payloads are small-molecule cytotoxics, while AOC payloads are nucleic acids designed to modulate gene expression rather than kill cells. AOCs are therefore more suitable for chronic diseases requiring long-term correction of genetic dysfunction.

What diseases can be treated using AOC technology?

AOCs are currently in clinical development for neuromuscular, oncological, and CNS disorders, with expanding research into metabolic and autoimmune diseases. Their modular design allows rapid adaptation to new targets.

What are the main technical challenges in AOC development?

Key challenges include efficient intracellular delivery, endosomal escape, and maintaining conjugate stability without impairing antibody or ASO function. Our proprietary linker chemistry and analytical platforms are specifically designed to address these issues.

Can you provide end-to-end AOC development services?

Yes. BOC Sciences offers complete AOC development solutions, covering antibody and ASO design, conjugation, analytical testing, process scale-up, and regulatory CMC support - from early discovery to GMP manufacturing readiness.

Ready to unlock the next generation of targeted RNA therapeutics?

Partner with BOC Sciences to accelerate your Antibody-ASO Conjugate (AOC) innovation with our integrated bioconjugation platform, advanced conjugation chemistry, and expert analytical support. We help you transform scientific ideas into high-quality, reproducible AOC solutions that deliver precision, stability, and performance. Contact us today to learn how our customized AOC development services can power your next discovery in RNA-based drug design.

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