Next-Generation In Vitro Platforms for ALS Drug Development & Target Validation

This workshop will explore the latest in vitro tools and model systems used to advance ALS drug discovery and target validation. Participants will discuss how human-derived cell models, induced pluripotent stem cells (iPSCs), and advanced 3D neuronal cultures are being leveraged to study disease mechanisms, screen therapeutic candidates, and identify biomarkers. The session will highlight best practices for experimental design, reproducibility, and translational relevance, as well as emerging technologies that enhance the predictive power of in vitro studies. Attendees will gain practical insights into selecting and applying these tools to accelerate ALS research and development.

Key Discussion Points

  • Overview of current in vitro models used in ALS research, including iPSC-derived motor neurons and glial co-cultures
  • Strategies for using in vitro systems to validate targets, test small molecules, genetic therapies, and combination approaches
  • Approaches to improve reproducibility, scalability, and translational relevance in ALS in vitro studies
  • Emerging technologies and high-content screening platforms to accelerate discovery and preclinical testing