Functional motor neurons from patient iPSC lines to support ALS research and drug discovery

The Modelling ALS with iPSC-Derived Cells brochure from Axol Bioscience introduces a comprehensive set of iPSC-derived motor neurons for modeling amyotrophic lateral sclerosis (ALS) in vitro. With no cure and limited treatment options, ALS research demands scalable, human-relevant models that reflect patient-specific phenotypes.

Axol’s axoCells™ ALS Toolbox includes motor neurons from six donor lines, both unaffected and ALS-affected (C9orf72, SOD1, TDP43 mutations). These cells are extensively characterized for morphology, marker expression, electrophysiology, and functional assays.

Key highlights include:

• Motor Neuron Characterization
  • Assay-ready in 10 days using Axol’s Motor Neuron Accelerator
  • Express key markers: HB9, ChAT, TUJ1, MAP2, LIM3
  • ALS-derived neurons show hyperexcitability, reduced synchronization, and altered burst dynamics
  • Validated on MEA and IncuCyte® platforms
• Disease Phenotypes Captured
  • ALS motor neurons exhibit irregular burst firing, higher burst rates, and lower synchronization
  • Phenotypes validated across multiple manufacturing runs for reproducibility
• Donor Lines Included
  • Unaffected: ax0076, ax0078
  • ALS: ax0074 (C9orf72), ax0079 (TDP43), ax0735 (SOD1)
  • C9orf72 Carrier: ax0073 (asymptomatic sibling of ax0074)

.Axol’s ALS Toolbox supports scalable, reproducible, and clinically relevant research into neurodegeneration.

Featured Axol Products:

• axoCells™ ALS Motor Neurons
  • ax0074 (C9orf72), ax0079 (TDP43), ax0735 (SOD1), ax0073 (C9orf72 carrier), ax0076, ax0078
• Motor Neuron Kits: Include media, supplements, and Accelerator
• axoServices™: Custom differentiation, assay development, and compound screening