Gene Therapy CRO Services
At Cyagen, we streamline your gene therapy pipeline—from AI-driven target discovery and AAV vector design to disease model generation and in vivo efficacy testing. Our integrated platform empowers translational research with speed, precision, and scientific confidence.
Visualize Your Path to Gene Therapy Success
Navigate every stage of your gene therapy program with clarity and confidence. Our
integrated workflow guides you from target discovery through vector engineering, model generation, and efficacy
validation—ensuring scientific continuity at every step.
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Predict. Validate. Accelerate.
AI-enabled discovery of therapeutic targets and pathogenic mutations.
Cyagen integrates advanced AI modeling with our proprietary rare disease database
(RDDC) to predict pathogenic gene mutations, design relevant models, and validate therapeutic
targets. Our platform bridges bioinformatics and wet-lab validation to de-risk and fast-track gene
therapy pipelines.
AI-Based Variant Prediction
Predict high-risk pathogenic variants using deep learning.
Model-Based Target Validation
Validate targets via custom cellular and animal models.
Rare Disease Data Center (RDDC)
Access curated mutation-disease databases for therapy design.
Integrated Screening Tools
Visualize mutation effects and explore RNA splicing outcomes.
AI-Based Variant Prediction
Uncover Disease-Causing Mutations with Predictive Precision
Evaluate the pathogenic potential and splicing consequences of genetic
variants using our AI-powered tools – Pathogenicity Predictor and RNA Splicer. These tools
leverage curated datasets from diseases like Duchenne Muscular Dystrophy (DMD) to identify
fun
Figure 1. Visualization from Cyagen’s RNA Splicer tool
showing splicing impact prediction of a pathogenic DMD variant.
Model-Based Target Validation
Validate Functional Impact Using Custom Cell and Animal Models
Translate predicted targets into experimental systems. Cyagen offers
KO, KI, and overexpression models—available in both rodent and cellular platforms—to confirm
variant pathogenicity and assess gene therapy responsiveness in vivo and in vitro.
Rare Disease Data Center (RDDC)
A Curated AI-Ready Database for Rare Disease Research
RDDC is a centralized hub for rare disease discovery, integrating
gene-disease associations, variant annotation, epidemiological data, and model repositories.
Researchers can identify targetable mutations and associated models for over 3,000 rare
conditions—all with AI-powered filters.
Integrated Screening Tools
Visualize Genotype-Phenotype Linkages in Seconds
Interact with gene, mutation, and model data via our visual dashboards
and natural language tools. From variant prioritization to model recommendation, our
screening platform simplifies early-stage decision-making for gene therapy programs.
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Design. Deliver. Transform.
AI-enhanced AAV vector engineering, high-titer packaging, and in vivo
validation—all in one place.
Cyagen’s AAV service platform supports the full cycle of vector development—from
AI-optimized capsid selection and cloning to customized packaging, purification, and QC validation.
Our inventory includes both off-the-shelf AAV serotypes and proprietary capsid variants for brain,
eye, and systemic delivery. Backed by validated in vivo and in vitro data, we ensure reliable
performance in your gene therapy research.
AI-Assisted AAV Capsid Design
Customize AAV serotype and cassette for your target tissue.
High-Titer AAV Production
Scalable production of high-quality AAVs with rigorous titering
and purity validation.
Ready-to-Ship AAVs
Access validated AAVs for common gene therapy applications –
shipped globally.
In Vitro & In Vivo Validation
Ensure precise delivery into target tissues with expert-guided
stereotaxic or systemic injection.
AI-Assisted AAV Capsid Design
Customize Capsids for Precise Targeting and Tissue Penetration
Leverage our AI-trained models to design capsids with enhanced
targeting for CNS, ocular, or hepatic delivery. Our proprietary variants (e.g., PM167,
PM170) demonstrate superior infectivity and organ selectivity—validated by in vivo data in
C57BL/6J mice.
Figure 2. In vivo fluorescence imaging showing enhanced
CNS tropism of PM167 and PM170 compared to PHP.eB.
High-Titer AAV Production Services
Customize Capsids for Precise Targeting and Tissue Penetration
Using triple transfection and advanced purification protocols, Cyagen
provides high-purity, high-titer AAVs with customizable serotypes and payloads. Our QC
ensures >95% purity and endotoxin <10 EU/ml—delivered in as fast as 2 weeks.
Figure 3. Fluorescence microscopy showing high
infection efficiency in 293T cells by AAV2-GFP at different MOIs.
Ready-to-Ship AAVs
Validated AAV Serotypes and AI-Optimized Variants
Access off-the-shelf AAV products for immediate research needs. Cyagen
offers standard serotypes and proprietary variants (e.g., PM077, PM021, PM054) with
validated expression in CNS and ocular tissues. Each lot is quality-controlled and
pre-tested for infectivity.
Figure 4. Comparative analysis of retinal layer
penetration by AAV2-WT vs. PM021 and PM054. PM054 shows 10x improved retinal
penetration.
In Vitro & In Vivo Validation
Verify Expression Before Preclinical Evaluation
We test vector efficiency across tissues using fluorescence, qPCR, and
functional phenotyping. AAV variants like PM077 and PM054 show enhanced ocular penetration,
validated via retinal layer imaging post-injection.
Figure 5. Frozen section of mouse eye tissue shows
superior retinal transduction by PM054 compared to AAV2-WT.
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Build. Validate. Translate.
Custom cellular and animal models for functional validation of gene therapy
targets.
Cyagen’s model generation platform empowers researchers to evaluate therapeutic
candidates in biologically relevant systems—from in vitro transduction assays to
disease-representative rodent models. We offer precise genome engineering for KO, KI, and
overexpression models, paired with comprehensive histological and biomarker validation to ensure
translational relevance for preclinical studies.
In Vitro Functional Assays
Validate gene function and transduction efficiency in cellular
systems.
Rodent Model Generation
Create disease-relevant KO, KI, and overexpression models for
preclinical research.
Histology & Biomarker Analysis
Confirm tissue-level changes and biomarker profiles with
precision.
In Vitro Functional Assays
Test Transgene Effects in a Controlled Cellular Environment
Use our optimized cell-based platforms to evaluate AAV-mediated
transduction efficiency, protein expression, and cell viability. Ideal for early-stage
screening before in vivo validation.
Rodent Model Generation
Validate Gene Function and Therapeutic Potential In Vivo
Establish knockout, knockin, or overexpression mouse models tailored
to
your gene of interest. Available on immunodeficient, humanized, or tissue-specific
backgrounds.
Histology & Biomarker Analysis
Uncover Biological Impact at the Tissue and Molecular Level
Perform detailed pathological analysis, including H&E, IHC/IF, and
biomarker profiling (e.g., dystrophin, inflammation, fibrosis markers) to confirm
therapeutic response.
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Deliver with Precision. Validate with Confidence.
Expert-guided CNS drug delivery to support gene therapy research.
Cyagen’s preclinical pharmacology team offers in vivo drug delivery services
tailored for CNS-targeted gene therapy research. From stereotactic injections into precise brain
regions to systemic administration methods, our experts ensure accuracy and reproducibility through
standardized procedures and post-delivery validation. Backed by imaging and biomarker readouts, we
help researchers confirm delivery success and downstream therapeutic effects.
Stereotactic Brain Injection
High-precision delivery to defined brain regions using 3D
coordinate systems.
Intrathecal Injection
Direct administration into cerebrospinal fluid (CSF) via lumbar
puncture or cisterna magna.
Intravenous (IV) Injection
Systemic delivery allowing broader biodistribution and tissue
transduction.
Additional Routes
Intramuscular, subcutaneous, intranasal, intraperitoneal, and more
– based on research goals.
Case Highlight
Example 1 – Precise Brain Targeting via Stereotactic
Injection
- Substance: AAV5-CAG-EGFP, AAV9-CAG-EGFP, PM227-CAG-EGFP
- Method: Stereotactic injection
- Target Site: Bilateral striatum
- Validation: EGFP expression observed 4 weeks post-injection via native fluorescence imaging, confirming successful localized brain transduction.
Figure 6. Representative coronal brain sections from mice
stereotactically injected with AAV5, AAV9, or PM227 vectors targeting the striatum. EGFP
signal (green) shows transgene expression; DAPI (blue) labels nuclei. Merge images
highlight co-localization. Brain atlas reference indicates targeting region.
Example 2 – Systemic Administration for Brain-Wide Gene
Delivery
- Substance: AAV9, AAV-PHP.eB, PM228
- Method: Intravenous injection via tail vein
- Validation: Robust, brain-wide GFP signal detected via immunofluorescence 3 weeks after administration, confirming efficient transduction across brain tissues.
Figure 7. Sagittal brain sections showing widespread EGFP
expression following intravenous delivery of AAV9 WT, PHP.eB, and PM228 vectors. Green:
EGFP signal; Blue: DAPI-stained nuclei. Merge panels show broad transgene distribution
throughout brain parenchyma.
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Measure. Analyze. Optimize.
Multi-tiered in vivo and in vitro assessments to quantify therapeutic
efficacy.
Cyagen delivers a full suite of efficacy evaluation services to support gene
therapy development. Whether assessing vector performance, biomarker expression, immune response, or
behavioral outcomes, our platform ensures scientific rigor and translational relevance. Testing is
conducted in validated models using standardized assays across molecular, cellular, tissue, and
systemic levels.
Molecular & Cellular Assays
qPCR, western blot, ELISA, and IF to quantify transgene expression
and protein function.
Functional Cell Readouts
Assays for proliferation, apoptosis, migration, and cellular
response to therapy.
Behavioral Studies
Water maze, open field, and gait analysis to evaluate CNS recovery
or degeneration.
Ocular Phenotyping
Retinal flat mount, ERG, and fundus imaging for evaluating ocular
gene therapy.
Case Highlight
Example – ASO-Mediated SMN Restoration in SMA Model
- Model: B6-hSMN2 mouse
- Assay: Western blot and IHC (ChAT staining)
- Outcome: ASO-10-27 treatment led to increased SMN protein levels and significantly higher numbers of spinal motor neurons, indicating effective target engagement and phenotypic improvement in the SMA model.
Figure 8. Western blot analyses confirm increased SMN
protein levels in B6-hSMN2 mice after ASO-10-27 administration, indicating successful
gene regulation. Immunohistochemistry (ChAT staining) further shows an increased number
of spinal motor neurons following treatment.
Our Unique Advantages
One Partner, Full Pipeline
Streamline your entire gene therapy workflow—target discovery, AAV design, model generation, and validation—all in one place.
AI-Powered Discovery
Accelerate progress with AI-guided variant prediction, splicing analysis, and capsid optimization from our proprietary RDDC platform.
Human-Relevant Models
Access validated humanized and disease-specific models, including HUGO-GT™ mice tailored for gene therapy research.
Request a Preclinical CRO Services Consultation
Partner with Cyagen to advance your preclinical studies. Share your project goals with us and receive customized support.
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