ASO in vitro Screening Service
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Overview
In recent years, nucleic acid therapeutics—including ASOs, siRNAs, aptamers, miRNAs, and sgRNAs—have gained prominence due to their high specificity, simple design, and broad therapeutic potential, with the global market projected to exceed $11.2 billion by 2025 and ASOs leading in clinical success and sales. Since the first FDA-approved ASO drug in 1999, over 13 therapies targeting genetic disorders have been approved worldwide, highlighting rapid clinical progress and strong industry leadership. The development of ASOs requires the integration of diverse technologies, including high-throughput screening, chemical modification, delivery systems, and animal models, to generate effective preclinical candidates. This process necessitates breakthroughs in basic research and interdisciplinary collaboration.
| Generic Name | Indication | Developer | Approval Year |
| Fomivirsen | Cytomegalovirus retinitis | Ionis | 1998 |
| Mipomersen | Familial hypercholesterolemia | Ionis | 2013 |
| Defibrotide | Hepatic veno-occlusive disease | Gentium Srl | 2013 |
| Eteplirsen | Duchenne muscular dystrophy | Sarepta Therapeutics | 2016 |
| Nusinersen | Spinal muscular atrophy | Ionis | 2016 |
| Inotersen | Hereditary transthyretin-mediated amyloidosis | Ionis | 2018 |
| Volanesorsen | Familial chylomicronemia syndrome | Akcea Therapeutics | 2019 |
| Golodirsen | Duchenne muscular dystrophy | Sarepta Therapeutics | 2019 |
| Viltolarsen | Duchenne muscular dystrophy | Nippon Shinyaku | 2020 |
| Casimersen | Duchenne muscular dystrophy | Sarepta Therapeutics | 2021 |
| Tofersen | SOD1-mutated amyotrophic lateral sclerosis | Ionis | 2023 |
| Eplontersen | Transthyretin amyloidosis with polyneuropathy in adults | Ionis | 2023 |
| Imetelstat | Myelodysplastic syndrome in adults not eligible or resistant to erythropoiesis-stimulating agents | Geron | 2024 |
Creative Biogene stands at the forefront of antisense oligonucleotide research, offering comprehensive ASO in vitro screening services that bridge the gap between molecular design and therapeutic validation. Our state-of-the-art platforms, combined with deep expertise in RNA biology and oligonucleotide chemistry, deliver actionable insights for your next breakthrough therapy.
Understanding Antisense Oligonucleotides
ASOs represent a powerful class of therapeutic molecules that leverage precise Watson-Crick base pairing to modulate gene expression at the RNA level.
Primary Mechanisms
- RNase H-Mediated Degradation: ASO-RNA duplexes recruit RNase H, resulting in specific mRNA cleavage
- Steric Blocking: Physical obstruction of ribosome binding or assembly, preventing translation
- Splicing Modulation: Alteration of splice site recognition, affecting mRNA isoform distribution
- MicroRNA Inhibition: Sequestration of regulatory miRNAs to restore target gene expression
Comprehensive ASO In Vitro Screening Service Portfolio
We provide a full-spectrum, customizable ASO in vitro screening platform designed to accelerate your discovery and development process through rigorous, multi-dimensional evaluation.
ASO Efficacy Screening
Transform your ASO discovery pipeline with our systematic efficacy evaluation platform. Moving beyond computational predictions, we provide empirical validation across multiple cellular contexts to identify your most promising candidates.
Service Components
Standard cell lines (HEK293, HeLa, A549, HepG2) and disease-specific models (cancer, neuronal, immune cells)
Primary Cell Compatibility
Patient-derived cells, iPSC-differentiated models, and organ-on-chip systems
Creative Biogene provides comprehensive ASO efficacy evaluation through precise RNA quantification (qRT-PCR, digital PCR), protein validation (Western blot, ELISA, mass spectrometry), and functional assays, including enzymatic and phenotypic analyses to assess biological impact.
Advanced Capabilities
Creative Biogene integrates advanced cellular analysis tools into its ASO screening platform, including high-content imaging for detailed subcellular localization and morphological profiling, live-cell monitoring to track ASO uptake and target engagement in real-time, and 3D culture systems such as organoids and spheroids to better replicate in vivo physiological conditions and enhance translational relevance.
ASO Specificity Analysis
Ensure therapeutic safety and selectivity with our comprehensive off-target assessment pipeline. Our multi-layered approach combines computational prediction with experimental validation to provide complete specificity profiles.
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Computational Analysis
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Genome-Wide Homology Screening: Identification of potential off-target sequences
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Thermodynamic Modeling: Prediction of binding stability and selectivity
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Pathway Impact Assessment: Systems-level analysis of potential downstream effects
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Experimental Validation
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Transcriptome Profiling: RNA-seq analysis to detect unintended gene expression changes
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Proteome Analysis: Mass spectrometry-based protein expression profiling
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Functional Specificity Assays: Target-specific reporter systems and rescue experiments
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Toxicity Screening: Cell viability, apoptosis, and stress response evaluation
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Immune Activation Assessment: Cytokine profiling and innate immune response analysis
Mechanism of Action Studies
Deepen your understanding of ASO function with specialized mechanistic investigations:
- RNase H Dependency Analysis: Confirmation of degradation-based mechanisms
- Subcellular Localization: Trafficking and compartmentalization studies
- Binding Kinetics: Association and dissociation rate measurements
- Structure-Activity Relationships: Systematic modification analysis
Technology Platform

Laboratory Infrastructure
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Automated Liquid Handling: Hamilton and Tecan systems for high-throughput processing
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Analytical Instrumentation: LC-MS/MS, capillary electrophoresis, and NMR spectroscopy
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Biosafety Compliance: BSL-2 certified facilities with full environmental monitoring
Quality Assurance

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ISO 9001:2015 Certified quality management system
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GLP-Compliant protocols for regulatory submissions
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Comprehensive Documentation with full audit trails
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Data Integrity measures, including electronic signatures and version control
Why Choose Creative Biogene
- Scientific Expertise: Creative Biogene brings over 15 years of oligonucleotide research experience, led by a team of PhD-level scientists with deep expertise in antisense oligonucleotides (ASOs). Our collaborative approach ensures continuous scientific support throughout every stage of your project.
- Technical Strength: We utilize proprietary protocols optimized for sensitivity and reproducibility, offering flexible study designs to accommodate varied research goals and scaling seamlessly from pilot projects to high-throughput campaigns. Our team also has strong regulatory experience to support IND filings and clinical trial applications.
- Global Service Standards: With international cold-chain logistics, 24/7 project tracking, and multilingual support in English, Chinese, Japanese, and European languages, Creative Biogene guarantees a responsive and efficient experience—backed by transparent, competitive pricing.
Contact our scientific team today to discuss your specific requirements and receive a customized proposal within 24 hours.
FAQ
Q1: How do you ensure consistent transfection across different cell types?
A1: We employ cell-type-specific optimization protocols using multiple delivery methods, including lipofection, electroporation, and nanoparticle systems. Each experiment includes transfection controls, efficiency markers, and standardized protocols validated for reproducibility.
Q2: What primary cell types can you accommodate?
A2: We regularly work with primary hepatocytes, neurons, cardiomyocytes, immune cells, and patient-derived samples. Our team can optimize protocols for specialized primary cells and develop custom co-culture systems to model tissue-specific environments.
Q3: How comprehensive is your off-target analysis?
A3: Our specificity assessment includes computational homology analysis, experimental transcriptome profiling (RNA-seq), functional validation assays, and immunogenicity screening. We provide both sequence-based predictions and experimental confirmation of specificity.
Q4: What format do you provide results in?
A4: You receive a comprehensive technical report including experimental design, raw data files, statistical analysis, graphical summaries, and expert interpretation. Data is provided in multiple formats suitable for publication, patent filing, or regulatory submission.
Q5: Do you offer follow-up optimization services?
A5: Yes, based on screening results, we can provide ASO sequence optimization, delivery system enhancement, and mechanism-of-action studies to further develop your lead candidates.
* For research use only. Not intended for any clinical use.