AI-Powered AAV Discovery
Accelerate your AAV vector innovation with AI-enhanced capsid design, predictive targeting, and validated in vivo performance for CNS and ophthalmic applications.
Explore AI-driven Breakthroughs in AAV Engineering
Traditional AAV capsid engineering faces critical limitations — small library capacities, low success rates,
and time-consuming iterations. Cyagen’s AI-powered discovery platform leverages deep learning and predictive
modeling to redefine AAV optimization, achieving unlimited library exploration, higher targeting accuracy,
and dramatically shorter development timelines.
Figure 1. Comparison of traditional vs. AI-driven AAV directed
evolution processes:
reducing cycles from 3–6 to 1–2 and timelines from >3 years to ~1 year.
Our AI-Enhanced AAV Discovery Workflow
By integrating intelligent mutagenesis, AI prediction, and high-throughput validation,
our workflow accelerates the generation of customized AAV capsids optimized for specific therapeutic needs.
Figure 2. Cyagen’s AI-driven AAV discovery workflow combines targeted
mutagenesis, deep learning algorithms, and in vivo validation to fast-track capsid selection.
AI-Driven Discovery of Next-Generation AAV Variants
Our AI-powered platform accelerates AAV discovery by predicting capsid variants with
enhanced transduction efficiency, tissue specificity, and reduced off-target effects – supporting faster
therapeutic development across neuroscience and ophthalmology.
{{ item.title }}
{{static_tabs[0].title}}
At Cyagen, we offer advanced in vivo drug delivery and phenotyping services tailored for
ophthalmic
studies. From precise ocular
injections to high-resolution imaging and vision behavior analysis, our platform provides
comprehensive
capabilities to evaluate
drug efficacy and safety. Supported by expert personnel and cutting-edge instrumentation, we
ensure
consistent, accurate, and
reproducible preclinical results.
| Group | Variant | Hippocampus | Cortex | Corpus Callosum | Midbrain | Spinal Cord | Liver |
|---|---|---|---|---|---|---|---|
| Wild-Type Control | AAV9 | 1 | 1 | 1 | 1 | 1 | 1 |
| Positive Control | AAV9.PHP.eB | 13.2 | 17.3 | 12.8 | 20.2 | 18.7 | 0.16 |
| AI Variant 1 | PM167 | 18.5 | 19.2 | 16.4 | 23.2 | 24.4 | 0.25 |
| AI Variant 2 | PM170 | 8.6 | 13.2 | 5.9 | 25.3 | 19.7 | 0.14 |
Data Validation Highlights:
- Bioluminescent imaging confirmed >2x and >1.5x stronger CNS expression for PM167 and PM170, respectively, versus PHP.eB.
- Frozen tissue sections (EGFP) validated enhanced neuronal and spinal cord transduction.
- Liver tropism suppression confirmed via imaging and histological analysis.
Figure: CNS Targeting and Liver Tropism of AI-Optimized AAV9 Variants
Looking for AAV vectors with enhanced CNS targeting and minimized
off-target effects?
Case Examples
Case 1: Enhanced CNS Transduction
AI-designed AAV9 variants PM167 and PM170 showed significantly stronger brain transduction—PM167
exceeded PHP.eB by ~2×. EGFP staining confirmed broader expression across hippocampus, cortex,
midbrain, spinal cord, and more.
Figure 1. AI model prediction for CNS-targeting capability shows strong
correlation with experimental ground truth (PearsonR = 0.843, SpearmanR = 0.663).
Figure 2. In vivo bioluminescent imaging and quantification reveal that PM167
achieves ~2× and PM170 ~1.5× stronger brain transduction compared to PHP.eB.
Figure 3. EGFP fluorescence imaging of brain and spinal cord sections confirms
enhanced neuronal transduction with PM167 and PM170 variants compared to PHP.eB and
wild-type AAV9.
{{static_tabs[1].title}}
Our AI-powered platform has developed novel AAV2 variants with superior retinal
penetration and robust full-eye expression, supporting advanced ophthalmic gene therapy
development.
| Group | Variant | Retinal Penetration | Full-Eye Expression |
|---|---|---|---|
| Wild-Type Control | AAV2-WT | 0 | 1 |
| Positive Control | AAV2.7M8 | 1 | 2 |
| AI Variant 1 | PM077 | 3 | 10 |
| AI Variant 2 | PM021 | 1.5 | 12.5 |
| AI Variant 3 | PM054 | 10 | 15 |
Data Validation Highlights:
- In vivo bioluminescence and chemiluminescence assays showed up to 15x higher expression for PM054 compared to AAV2-WT.
- Retinal flat-mount imaging confirmed widespread transduction across all retinal layers.
- Penetration into photoreceptor cells significantly surpassed conventional AAV variants.
Figure: Retinal Penetration and Full-Eye Expression of AI-Optimized AAV2
Variants
Need next-generation AAV vectors optimized for retinal gene
therapy?
Case Examples
Case 1: High Viral Yield Prediction and Validation
Using an AI-based production prediction model with high accuracy (Pearson R =
0.929, Spearman R = 0.859), Cyagen successfully identified AAV2 variants with significantly
enhanced viral yields. Experimental validation confirmed that PM054 achieved approxima
Figure 4. Validation of the AAV2 production prediction model showing strong
correlation between predicted and actual yields.
Figure 5. Viral yield ratios of top AI-designed variants compared to AAV2-WT
and AAV2.7M8, demonstrating enhanced production.
Figure 6. Bioluminescence imaging results showing stronger full-eye luciferase
expression for PM077, PM021, and PM054 compared to controls.
Figure 7. Quantitative analysis of luciferase expression from homogenized eye
tissues, highlighting superior expression levels of AI variants.
Figure 8. Fundus fluorescent imaging showing widespread retinal infection
across PM077, PM021, and PM054-treated eyes.
Figure 9. Retinal section imaging showing extensive GFP expression from the
RGC layer through to the PRC layer for PM054 and related variants.
Our Unique Advantages
Proprietary Deep Learning Models
Our in-house AI algorithms predict tissue targeting, expression capacity, and vector tropism with high accuracy, validated by biological assays.
Extensive Variant Libraries
We offer multiple ready-to-use brain-targeting and ocular-targeting AAV variants, along with tissue-specific promoter collections for customized applications.
Integrated Gene Therapy CRO Services
From AI-guided capsid design to comprehensive preclinical validation, Cyagen supports a seamless continuum for your gene therapy development.
Request a Preclinical CRO Services Consultation
Partner with Cyagen to advance your preclinical studies. Share your project goals with us and receive customized support.
Cyagen values your privacy. We’d like to keep you informed about our latest offerings and insights. Your preferences:
You may unsubscribe from these communications at any time. See our Privacy Policy for details on opting out and data protection.
By clicking the button below, you consent to allow Cyagen to store and process the personal information submitted in this form to provide you the content requested.