Preclinical Efficacy Evaluation

Predict Clinical Outcomes for CNS and PNS Diseases and Disorders

Human-relevant disease models for central nervous system (CNS) and peripheral nervous system (PNS) diseases and disorders measure functional, electrophysiological responses in human-derived 3D neural tissues, providing predictive insights that better align with clinical outcomes.

Custom Disease Model Development for Efficacy Evaluations

CNS or PNS models can be tailored to reflect specific patient populations or disease phenotypes using our Custom Organoid Services. Whether evaluating therapeutic efficacy or exploring mechanistic insights, each model is optimized to match your compound’s target biology.

Disease Areas

Central Nervous System
Peripheral Nervous System
Rett Syndrome
Identifying effective therapeutics with human neural models
CNS-3D technology models disease-relevant neural activity using patient-derived iPSCs and matched isogenic controls to identify therapeutic candidates for Rett Syndrome. Organoids exhibit distinct electrophysiological signatures, with Rett samples showing irregular firing compared to the coordinated bursting in controls.

Proprietary analytics extract 67 functional metrics, synthesized into a Global Recovery score to rank treatment responses. Because functional activity clusters by mechanism of action, the technology also reveals how different therapies affect neural networks, offering clinically relevant insights with predictive power.
Functional signatures of CNS-3D Rett Syndrome organoids treated with Donepezil, showing dose-dependent rescue.
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CDKL5 Deficiency Disorder
High-throughput phenotypic screening for disease-modifying therapies
To identify novel therapeutics for CDKL5 Deficiency Disorder (CDD), a parallel screen is conducted using human neural organoids from both CDD patients and familial controls. An initial single-dose screen of 5,200 compounds yields 288 hits, which are rescreened to assess selectivity.

A follow-up 7-point dose-response assay refines the list to 22 top candidates across 15 biological targets. Comparing IC50 values between CDD and control organoids highlights compounds with selective potency in the disease model, linking their mechanisms to CDD-specific pathway disruptions.
CNS-3D Control and CKDL5 KO organoids share similar cellular ratios neurons (MAP2/green) and astrocytes (GFAP/magenta) but exhibit distinct functional signatures. CNS-3D CDKL5 KO organoids display network hyperexcitability.
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Chemotherapy-induced Peripheral Neuropathy
Differentiating effective from ineffective neuroprotective strategies
PNS-3D technology integrates human iPSC-derived sensory neurons and primary Schwann cells to model functional deficits associated with chemotherapy-induced peripheral neuropathy (CIPN).

The technology replicates neurotoxic responses, such as those induced by vincristine, and supports evaluation of neuroprotective strategies, including SARM1 inhibition. It enables detection of dose-dependent effects on axonal integrity and the onset of neurotoxicity, providing a powerful tool for assessing therapeutic efficacy and optimizing dosing strategies.
SARM1 inhibitor treatment effects over time across electrophysiology (left), neurite-growth metrics (middle), and the combination of both (right). While vincristine alone causes swift declines in function and morphology, NB-7 and WX-02-37 confer prolonged protection across endpoints compared to DSRM-3716, which mirrors the positive-control decline.
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Preclinical Efficacy Resources

Scientific Poster
6/10/2024
Identification of a Potential Therapeutic Compound for Rett Syndrome Using a Highly Homogenous Human iPSC-derived Cortical Organoid Screening Platform
Publication
6/27/2022
A Multiparametric Calcium Signal Screening Platform Using iPSC-Derived Cortical Neural Spheroids
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