Oligonucleotide Therapy CRO Services
From in vitro knockdown screening to in vivo survival outcomes—Cyagen delivers end-to-end CRO services tailored for ASO, siRNA, and precision gene modulation therapies.
Your End-to-End Path to Oligo Therapeutics
Cyagen’s platform is purpose-built for the unique demands of oligonucleotide
therapeutics—ensuring precise evaluation of ASO and siRNA drugs across molecular, cellular, and organismal
levels. Our full-cycle services span from early screening to in vivo validation, all within a unified system
that delivers consistency, reproducibility, and translational value.
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Silence. Confirm. De-risk.
Validate target modulation through molecular and functional assays.
Establish the efficacy of oligonucleotide drugs at the molecular level by
confirming their ability to reduce gene expression in vitro. Cyagen provides a full suite of
assays—including mRNA quantification and protein-level confirmation—to ensure your ASO, siRNA, or
gene-targeting construct hits the intended target with high specificity.
Cyagen’s in vitro knockdown validation services include:
- qPCR & RT-qPCR: Quantitative analysis of mRNA knockdown levels
- Western Blot / ELISA / IF: Confirm reduced protein expression
These assays offer the first checkpoint in confirming oligo activity before in
vivo studies begin.
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Replicate. Predict. Translate.
Use clinically relevant HUGO-GT™ models for oligo drug development.
Cyagen’s HUGO-GT™ humanized mouse models enable accurate evaluation of oligonucleotide therapies in
vivo. These models feature full human sequence replacement of key gene loci—ensuring compatibility
with ASO, siRNA, and CRISPR-based drugs. With validated models for SMA, ALS, Alzheimer’s disease,
RP, and more, we support precise pharmacodynamic assessments tailored to your therapy’s genetic
target.
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Deliver. Track. Optimize.
Evaluate compound uptake and tissue targeting across ICV, IV, and SC routes.
We provide tailored delivery strategies and quantitative analyses to evaluate ASO, siRNA, or other
oligonucleotide biodistribution in vivo.
- Supported delivery routes: Intracerebroventricular (ICV), intravenous (IV), and subcutaneous (SC) injection
- Quantitative assessment: Fluorescence imaging, RT-qPCR, and histological analysis
These tools ensure accurate evaluation of compound exposure and tissue
localization—critical for therapeutic optimization.
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Evaluate. Quantify. Validate.
Multilevel pharmacodynamic readouts—molecular, cellular, histological, and
functional—ensure robust efficacy assessment.
Cyagen’s PD platform offers a full range of validated assays to evaluate
oligonucleotide drug performance in vivo and in vitro. Whether quantifying SMN expression in SMA
models or assessing visual and motor function recovery, our customizable evaluation pipeline
delivers reliable, publication-ready data.
| Evaluation Type | Description |
|---|---|
| Molecular Assays | qPCR, Western blot, ELISA for gene and protein expression profiling. |
| Cellular Assays | Viability, apoptosis, and proliferation assays for assessing cellular response. |
| Histological Readouts | H&E, IHC, and immunofluorescence to evaluate tissue structure and target markers. |
| Functional Readouts | ERG and behavior tests (e.g., motor and cognitive assessments) for efficacy validation. |
Need Evaluation Support? Tell us about your study goals!
Our team will help design a tailored PD and safety evaluation plan.
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Case Highlight - ASO Efficacy in SMA Humanized Mouse Model
Restored SMN Expression & Improved Motor Neuron Survival
Treatment with ASO-10-27 significantly increased SMN protein levels and motor neuron
counts in B6-hSMN2 mice, as demonstrated by western blot and ChAT immunostaining.
Figure 1. ASO treatment increased SMN expression and motor neuron count in
B6-hSMN2 mice, confirmed by western blot and ChAT immunostaining after ICV and SC injection.
Extended Survival and Delayed Disease Onset
B6-hSMN2 mice receiving ASO-10-27 via ICV injection exhibited markedly improved
survival and delayed disease symptoms compared to untreated controls.
Figure 2. ASO-10-27 treatment improved survival and delayed disease symptoms in
B6-hSMN2 mice, with reduced toe necrosis and preserved tail integrity at 78 days
post-injection.
Our Unique Advantages
Humanized Models Optimized for ASO/siRNA
Access our exclusive HUGO-GT™ mouse models with full-length human gene replacement—ideal for evaluating ASO, siRNA, and gene modulation therapies targeting human-specific sequences.
Proven Preclinical Study Experience
Work with a team backed by successful case studies in oligonucleotide drug development across CNS, neuromuscular, and ophthalmic diseases.
Integrated CRO Platform from In Vitro to In Vivo
Seamlessly transition from in vitro knockdown assays to in vivo pharmacology and biodistribution—ensuring consistency and scientific rigor at every step.
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