Neuro developmental disorders

Our platform offers a comprehensive collection of synaptopathy and genetic epilepsy iPSC models designed to accelerate preclinical discovery. This unique library includes patient‑derived iPSC lines, isogenic controls, and gene‑edited control lines carrying patient‑specific mutations or targeted gene deletions.

Upon differentiation into excitatory and inhibitory iNeurons, these models consistently demonstrate disease‑relevant alterations in neuronal network behavior and synaptic function when compared to their isogenic controls.

Through systematic functional characterization, we have built an extensive, high‑quality reference dataset. This enables fast, reliable, and reproducible preclinical efficacy data  to evaluate drug candidates with confidence and reduce translational risk.

Available IPSC disease models

  • Patient and isogenic control lines of Synaptopathies as STXBP1-related disorder
  • Patient and isogenic control lines of genetic epilepsy disorders
  • Customized gene-edited lines with patient-specific mutations or targeted gene deletions
  • Pharmacological assays of epilepsy

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Characterizations

We have fully characterized excitatory and inhibitory iNeurons derived from patient‑specific and isogenic control iPSC lines across multiple neurodevelopmental disorder models. This characterization spans neuronal network dynamics, single‑cell electrophysiology, live microscopy of single synapse function, molecular expression, and detailed morphology, capturing more than 90 quantitative parameters.

With these comprehensive datasets established, our models are fully validated and ready for multiscale compound testing within our integrated screening pipeline. We offer a broad suite of assays, ranging from high‑content functional readouts to in‑depth profiling of expression patterns and cellular morphology. All assays can be fully customized to meet specific requirements within preclinical pipelines, enabling efficient and precise discovery of compound effects at the network, cellular, and synaptic levels.

Example dataset of functional characterization of patient and isogenic control IPSC-derived iNeurons according to automated pipeline developed by Neurospector.

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