Characterization of Human iPSC-Derived Motor Neuron Disease Model for ALS Drug Discovery

The poster Characterization of Human iPSC-Derived Motor Neuron Disease Model for ALS Drug Discovery presents Axol Bioscience’s development of a physiologically relevant ALS model using iPSC-derived motor neurons from genetically diverse donors.

Key highlights include:

• Donor Diversity:
  • 6 donor lines: 2 unaffected, 1 C9orf72 carrier (unaffected sibling), 3 ALS patients (C9orf72, SOD1, TDP43)
• Morphological Characterization:
  • Brightfield imaging shows distinct neurite outgrowth and network formation
  • ALS lines exhibit smaller, irregular clusters and fibrous neurites
• Marker Validation:
  • TUJ1 expression confirmed via ICC across all lines
• Functional Assays:
  • Spontaneous Neuronal Activity (SNA) via IncuCyte® S3
    • ALS lines show increased burst rate and reduced synchrony
  • MEA Analysis via Axion Maestro Pro
    • Confirms hyperexcitability and loss of synchronous firing in ALS lines
• Phenotypic Differences:
  • Each ALS mutation displays distinct activity profiles
  • Supports patient stratification for therapeutic screening
• Manufacturing Consistency:
  • Multiple production runs confirm reproducibility
  • Functional QC integrated into standard workflows

This model enables high-throughput ALS drug screening with mutation-specific insights and robust functional validation.

Featured Axol Products:

• axoCells™ Motor Neurons – Unaffected Donors – ax0076, ax0078
• axoCells™ Motor Neurons – ALS (C9orf72) – ax0074
• axoCells™ Motor Neurons – ALS (SOD1) – ax0735
• axoCells™ Motor Neurons – ALS (TDP43) – ax0079
• axoCells™ Motor Neurons – C9orf72 Carrier (Unaffected) – ax0073
• axoCells™ Motor Neuron Accelerator – ax0179