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Integrated RNA Therapeutics

One-Stop
ASO Development Services

From Sequence Design to Preclinical Support. We provide comprehensive solutions covering ASO design, synthesis, modification, conjugation, analysis, and in vivo/in vitro validation.

Explore Solutions

ASO Technology Overview

Antisense Oligonucleotides (ASOs) are short, synthetic, single-stranded oligodeoxynucleotides that can alter RNA expression. They operate primarily through mechanisms such as RNase H-mediated degradation or Steric blocking to modulate splicing.

Key Indications: Genetic disorders, Oncology, Neurodegenerative diseases (e.g., SMA, DMD), and Viral infections.

Why Choose One-Stop Development?

  • Reduce project management complexity
  • Accelerate R&D efficiency
  • Ensure technical continuity and quality consistency
ASO Research

Who We Serve

Our flexible platform adapts to the needs of diverse partners, from agile startups needing full incubation to pharma giants requiring specific module support.

Biotech Companies

Pharmaceutical Enterprises

Academic & Research

Startups (Incubation)

One-Stop ASO Solutions

Comprehensive modular services tailored to your specific drug development needs.

ASO Design
ASO Design & Sequence Optimization

We utilize advanced algorithms and empirical rules to design high-potency sequences while minimizing off-target risks.

  • ✓ Target Analysis: Comprehensive strategy formulation based on gene targets.
  • ✓ Algorithm Design: Proprietary algorithms combining empirical rules for optimal sequence selection.
  • ✓ Risk Assessment: In silico Off-target risk evaluation.
  • ✓ Screening: Sequence screening and optimization suggestions.
ASO Synthesis
ASO Synthesis & Chemical Modification

Flexible synthesis scales and diverse modification options to enhance stability and binding affinity.

Synthesis Capabilities

  • • Gapmer ASO
  • • Steric-blocking ASO
  • • Scale: mg to gram level

Chemical Modifications

  • • Phosphorothioate (PS)
  • • 2′-OMe / 2′-MOE
  • • LNA / cEt (Bridged Nucleic Acids)
Conjugation
ASO Conjugate Development

Targeted delivery solutions to improve tissue specificity and cellular uptake.

GalNAc-Conjugated Antibody-Conjugated Fluorophore-Conjugated Polymer-Conjugated

Services Include: Definition of conjugation purpose, matching appropriate tissue/indications, and fully customized development capabilities.

In Vitro Screening
In Vitro Screening

High-throughput cellular assays to rapidly identify the most potent lead candidates.

  • ✓ Cell Models: Wide range of cell lines and primary cell models.
  • ✓ Efficacy: mRNA knockdown quantification (qPCR, bDNA) and Protein level analysis.
  • ✓ Safety: Cytotoxicity assays (MTT, CCK-8, LDH).
In Vivo Evaluation
In Vivo Efficacy & Safety Evaluation

Comprehensive animal studies to validate therapeutic potential in a living system.

  • Animal Model Support
  • PK / PD Studies
  • Tissue Distribution Analysis
  • Safety & Tolerability Assessment
ASO Delivery
ASO Delivery Strategies

Overcoming cellular uptake barriers is the key to ASO success. We offer diverse delivery platforms to ensure your ASO reaches its target.

  • ✓ Lipid Nanoparticles (LNPs): Optimized formulations for hepatic and extra-hepatic delivery.
  • ✓ Exosome-Mediated: Natural carriers for low immunogenicity and high efficiency.
  • ✓ Peptide Carriers: Cell-penetrating peptides (CPPs) to enhance endosomal escape.
Off-Target Detection
Off-Target Detection and Analysis

Ensuring the safety and specificity of your ASO therapeutics through advanced genomic analysis.

Transcriptome-wide Analysis (RNA-Seq)

Unbiased detection of gene expression changes to identify unintended hybridization sites.

In Silico Prediction Validation

Experimental verification of potential off-target sites predicted by algorithms.

Development Workflow

01
Target & Sequence Design
02
Synthesis & Modification
03
Conjugation (Optional)
04
In Vitro Evaluation
05
In Vivo Evaluation
06
Bioanalysis & Data

Bioanalysis & QC Support

Bioanalysis & Quality Control

  • ASO Purity & Integrity Analysis (LC-MS / HPLC)
  • Stability Studies (Serum/Plasma)
  • Quantitative Bioanalysis in Biological Matrices

Project Delivery

  • Full Technical Reports & Methodology Summary
  • Data Integration & Interpretation
  • Preclinical Study Support (IND-enabling)

Why Partner with Us?

One-Stop Platform

Seamless integration from design to in vivo proof-of-concept.

Expertise

Rich experience in complex modifications and conjugation.

Customization

Tailored strategies, not just standard "off-the-shelf" kits.

Quality System

Rigorous QC systems ensuring data reliability.

Frequently Asked Questions

The timeline varies depending on the complexity of the project and specific requirements. Generally, a comprehensive cycle from In Silico Design to In Vitro Validation takes approximately 6 to 12 weeks. This includes roughly 1-2 weeks for bioinformatics analysis and sequence design, 2-3 weeks for synthesis and purification (depending on scale and modifications), and 4-6 weeks for cell-based screening and efficacy validation (qPCR/Western Blot). For In Vivo studies, timelines will be determined based on the animal model and dosing regimen selected.
We offer a comprehensive library of chemical modifications to enhance nuclease resistance and binding affinity. Our capabilities include backbone modifications like Phosphorothioate (PS), sugar modifications such as 2'-O-Methyl (2'-OMe), 2'-O-Methoxyethyl (2'-MOE), and Locked Nucleic Acids (LNA) or cEt. We can also synthesize Gapmer designs optimized for RNase H recruitment or steric blocking oligos for splice modulation. Custom conjugation services are also available upon request.
Yes. While we provide GalNAc conjugation for highly efficient liver targeting, we also specialize in delivery strategies for extra-hepatic tissues. This includes the development of Lipid Nanoparticles (LNPs) for systemic delivery, Antibody-Oligonucleotide Conjugates (AOCs/ARCs) for cell-specific receptor targeting (e.g., muscle or tumor tissues), and Exosome-mediated delivery systems which offer low immunogenicity and the ability to cross biological barriers like the blood-brain barrier (BBB).
We employ a multi-layered approach to mitigate off-target risks. First, we utilize proprietary algorithms for In Silico prediction to filter out sequences with high homology to non-target genes. Following synthesis, we conduct experimental validation using Transcriptome-wide RNA-Seq to monitor global gene expression changes and identify potential hybridization-dependent off-target effects. We also perform standard cytotoxicity assays (MTT/CCK-8) and immunogenicity assessments to ensure the safety profile of your lead candidates.
We adhere to strict confidentiality protocols to protect your intellectual property. All data, sequences, and experimental results generated during the project are the sole property of the client. We are fully prepared to sign a Non-Disclosure Agreement (NDA) prior to any technical discussions or data exchange. Our secure data management systems ensure that your research information remains private and is only accessible to authorized personnel working on your project.

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