Applications

CNS Toxicity

Predict CNS toxicity risk by measuring drug-induced changes in neuronal network activity. In small molecule and ASO toxicity studies, CNS-3D Brain Organoids have outperformed animal models and 2D assays, supporting more confident assessment of drug-induced neurotoxicity.

Detect Functional CNS Toxicity

Central nervous system (CNS) toxicity drives 25% of safety-related drug development failures, in part because conventional preclinical models can miss functional disruption that later translates into clinical safety issues. Animal studies rely on indirect behavioral endpoints, while 2D cultures and viability assays lack the human 3D neural network biology needed to assess drug effects on neural activity.

CNS-3D Technology addresses this translational gap by measuring drug-induced functional changes in human 3D cortical tissue. In small molecule seizure risk and ASO neurotoxicity studies, CNS-3D outperformed alternative models, achieving 83% sensitivity / 89% specificity and 92% sensitivity / 92% specificity, respectively. By combining human cells, multicellular complexity, and AI-based neurotoxicology prediction, CNS-3D supports more accurate toxicity prediction and candidate rank ordering across diverse drug modalities.

Advantages

Confident Neurotoxicity Prediction

By measuring how compounds alter neuronal network activity in human 3D tissue, CNS-3D supports more confident neurotoxicity prediction across therapeutic modalities.

Human 3D 
CNS Biology

Assess drug effects in human iPSC-derived cortical organoids with multicellular complexity

Functional
Toxicity Detection

Detect neurotoxic effects when drugs disrupt neuronal network activity—before damage appears in viability-only assays.

Broad Modality
Coverage

Evaluate neurotoxicity risk across diverse modalities, including small molecules, ASOs, and gene therapy vectors.

Reproducible
Performance

Standardized organoid production and robust QC drive consistent results from experiment to experiment.

Use Case

Small Molecule Toxicity

Accurately identify seizurogenic small molecules while minimizing the risk of eliminating clinically safe drugs. CNS-3D Brain Organoids combine functional neural activity with AI-based prediction to assess clinical seizure risk with high confidence. Across 66 small molecules with known clinical outcomes, CNS-3D achieved 83% sensitivity and 89% specificity.

View Application Note

Sensitivity and specificity comparison for small molecule seizure risk prediction, demonstrating superior performance of CNS-3D versus alternative models.

Use Case

ASO Toxicity

In a study of 24 ASOs, CNS-3D Brain Organoids outperformed sequence-based prediction and 2D calcium assays, achieving 92% sensitivity and 92% specificity. By measuring functional activity in human 3D neural tissue, CNS-3D captures toxicity signals that sequence-based and 2D approaches can miss. Beyond identifying ASOs as toxic or safe, CNS-3D captures gradations of neurotoxicity, enabling more precise candidate rank ordering.

View Application Note

CNS-3D ASO neurotoxicity predictions compared with sequence-based and 2D calcium assay results, showing improved sensitivity and specificity across 24 ASOs.

Use Case

AAV Toxicity

CNS-3D Brain Organoids detect functional disruption at doses below overt viability loss, enabling more sensitive safety assessment than viability assays alone. By integrating expression, neuronal network activity, and viability assessment, CNS-3D helps identify vectors that balance transduction efficiency with functional safety.

View Application Note

Functional calcium imaging detects AAV-associated disruption of neuronal network activity before ATP-based viability loss, supporting more sensitive assessment of vector impact than viability endpoints alone.

Offerings