Question 1

What efficacy applications can 28bio support?

Accepted Answer

We support gene therapy vector evaluation, neuroinflammation, myelination, Alzheimer’s disease, and peripheral neuroprotection. Studies can be designed to evaluate whether candidates improve disease- or injury-associated phenotypes using functional nervous system readouts with complementary molecular, imaging, and viability endpoints. Contact us to discuss your disease area of interest.

Question 2

How are disease states modeled?

Accepted Answer

We support induced injury models, patient-derived iPSC-based models, and CRISPR-engineered approaches where appropriate. CNS models can incorporate induced, patient-derived, or engineered disease biology to study functional phenotypes in brain organoids. PNS models combine iPSC-derived sensory neurons with primary Schwann cells, enabling tailored approaches such as sensory neuron gene editing, injury induction, or disease-relevant assay design.

Question 3

How is drug efficacy measured in CNS and PNS organoid models?

Accepted Answer

Efficacy is measured by assessing whether a candidate improves disease-, injury-, or treatment-induced changes in nervous system function. CNS studies use functional neural activity readouts such as calcium imaging, while PNS studies can assess peripheral nerve function. These functional endpoints can be paired with viability, imaging, gene expression, protein, or biomarker readouts to connect functional improvement with biological mechanism.

Question 4

What makes 28bio efficacy data more actionable than conventional preclinical models?

Accepted Answer

Conventional models often rely on animal biology, simplified 2D cultures, or indirect endpoints that may not reflect human nervous system function. Our CNS and PNS organoid models combine human-derived tissue with functional readouts, helping researchers assess whether a candidate produces a measurable biological effect in a disease-relevant context before advancing into later-stage studies.

Question 5

Can 28bio support both early screening and deeper lead characterization?

Accepted Answer

Yes, study designs can be configured for different discovery stages, from higher-throughput hit identification to deeper profiling of selected leads. Early screens can prioritize broader compound coverage, while lead characterization can incorporate additional concentrations, functional metrics, viability, imaging, and molecular endpoints to distinguish promising candidates from those with limited efficacy or dose-limiting toxicity.

Question 6

How can researchers access 28bio efficacy models and studies?

Accepted Answer

Researchers can access assay-ready CNS-3D products for in-house studies or work with our service team for study design, execution, and analysis. Custom services are available for programs requiring disease-specific biology, including induced, patient-derived iPSC, and CRISPR-engineered disease modeling approaches. PNS-3D efficacy studies are offered through services.

Question 7

How scalable are CNS and PNS efficacy studies?

Accepted Answer

Efficacy studies can support the full drug development continuum, from early candidate screening to deeper functional characterization of lead therapies. CNS-3D Brain Organoids support studies from a single plate up to 20,000 samples per day in 24-, 48-, 96-, and 384-replicate formats, available as assay-ready products or through CNS Services. For PNS efficacy, PNS-2D supports scalable 96-well neuroprotection screening, while PNS-3D enables longitudinal functional assessment in a 24-well embedded electrode array format, with staggered cohorts available for larger studies.

Efficacy Assessment

Predict Neurological Disease Drug Efficacy

Efficacy Applications

Gene Therapy

Neuroinflammation

Myelination

Alzheimer’s Disease

Efficacy Products & Services

CNS-3D Brain Organoids

CNS-3D Induced Alzheimer’s Model

CNS-3D Myelinated Organoids

CNS-3D Inflammatory Organoids

CNS Services

Efficacy Resources

Frequently Asked Questions

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