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A human iPSC-based platform to screen therapeutics for ALS using assays covering disease- relevant readouts.​

Physiologically relevant ALS screening platform using human iPSC-derived motor neurons
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A human iPSC-based platform to screen therapeutics for ALS using assays covering disease- relevant readouts_ADPD_2026

This poster presents Ncardia’s human iPSC-based platform for modeling and screening amyotrophic lateral sclerosis, built around disease-relevant motor neuron assays that capture key pathological hallmarks of TDP-43 dysfunction.

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  • How CRISPR-engineered TDP-43 mutant motor neurons reproduce core ALS phenotypes, including TDP-43 mislocalization, aggregation, STMN2 reduction and mis-splicing.

  • High-content imaging and miniaturized assay formats that enable quantitative, scalable assessment of protein aggregation and molecular rescue.

  • Electrophysiological characterization by MEA, demonstrating altered network activity and functional deficits in mutant motor neurons.

  • Proof-of-concept data showing successful transduction and therapeutic rescue, including significant reduction of TDP-43 aggregation and improvement of functional readouts.

  • Why integrating molecular, imaging, and functional endpoints in a single human-relevant platform supports more predictive evaluation of ALS therapeutic candidates.

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Whether you are running early proof-of-concept studies or building a long-term ALS discovery strategy, Ncardia’s human iPSC-derived motor neuron platform provides a scalable, reproducible foundation for high-content, high-throughput screening and confident data-driven decisions.


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