Discovery Pharmacology for ALS: Models, Mechanisms, Biomarkers
Amyotrophic lateral sclerosis (ALS) remains one of the most active and complex areas of CNS drug discovery—driving demand for preclinical models that offer mechanistic clarity, translational relevance, and adaptability across therapeutic modalities. As novel strategies continue to emerge, researchers are advancing in vitro and in vivo tools to better model motor neuron disease biology, evaluate pharmacological response, and align clinical biomarker frameworks.
Join Stefan Ebner-Benke, PhD, Director In Vivo, Scantox Neuro, for Discovery Pharmacology for ALS: Models, Mechanisms, Biomarkers recorded on November 25, 2025, followed by a Q&A session.
This session will explore how in vitro and in vivo models are used to investigate key mechanisms in ALS—such as TDP-43 aggregation and mislocalization, SOD1-mitochondrial dysfunction and oxidative stress —and how these models are selected and adapted to support early discovery through candidate selection. The discussion will focus on practical considerations for study design, biomarker strategy, and aligning models with therapeutic modality and mechanism of action.
Key Learning Objectives
- Review core and emerging ALS models—considering key mechanisms like TDP-43, SOD1, mitochondrial degeneration and neuroinflammation—and how they support different stages of drug discovery.
- Understand how preclinical models are used to evaluate translational biomarkers, target engagement, and disease modification.
- Learn how to align model selection with program goals—from early screening to mechanism-of-action refinement.
- Explore study design variables including modality, administration route, drug target, CNS delivery strategy (e.g., intrathecal for ASOs), and treatment timing.
Who Should Attend
- Neuroscientists and discovery pharmacologists working in CNS drug development.
- Translational researchers focused on ALS.
- Biotech and pharma teams developing novel modalities for neurodegeneration
- Nonclinical consultants, CRO collaborators, and program strategists seeking fit-for-purpose models.
Meet The Presenter
Dr. Stefan Ebner-Benke brings extensive experience in the design, coordination, and execution of complex nonclinical studies supporting novel neuropharmacological discovery. As Director In Vivo at Scantox Neuro, he partners with biotech and pharmaceutical clients to translate early-stage CNS research into actionable data through scientifically rigorous and operationally robust study programs. His multidisciplinary background spans academic research, strategic consulting, and nonclinical operations, with a proven track record of driving excellence in preclinical study delivery. He holds a PhD in Molecular Biology from the University of Vienna, an MSc in Molecular Microbiology from the University of Graz, and an Executive MBA from California Lutheran University.