Human iPSC-Based ALS Screening Platform for Phenotypic Drug Discovery

This poster presents Ncardia’s human iPSC-derived motor neuron platform, designed to model key ALS disease phenotypes and enable high-throughput screening of therapeutic candidates in a physiologically relevant system. 

Download it now for a closer look at:

• How CRISPR-engineered TDP-43 mutant motor neurons replicate core ALS hallmarks, including protein mislocalization, aggregation, and downstream molecular effects 
  
• Quantitative assays measuring TDP-43 pathology, STMN2 reduction and mis-splicing, neurofilament-L secretion, and electrophysiological dysfunction 
• How complex co-culture systems with astrocytes enhance physiological relevance and capture disease-specific cellular interactions 
• Evidence that the platform can be miniaturized to 384-well format, supporting scalable, high-throughput compound screening 
• Proof-of-concept data showing successful gene therapy intervention, including reduced TDP-43 aggregation following treatment