SfN 2025 – A Human iPSC-Derived Motor Neuron Platform: Applications for Drug Discovery and ALS Research

Recreating ALS pathology in vitro to uncover tomorrow’s treatments

Amyotrophic lateral sclerosis (ALS) is a complex neurodegenerative disease with limited therapeutic options and a high clinical failure rate. To address this, we’ve developed a human iPSC-derived motor neuron platform, in collaboration with Axol Bioscience, with validated ALS-relevant assays reflecting key pathophysiological features, including mitochondrial dysfunction.

Our scalable platform enables high-throughput drug screening and target validation in 96- and 384-well formats. We demonstrate phenotypic differences between motor neurons from ALS patients and healthy donors and outline future directions using stressors to model multi-step disease progression.

Download the poster to see how this platform accelerates ALS drug discovery through functional, human-relevant models.