Christopher Shaw
Chief Scientific & Clinical Advisor AviadoBio
Professor Shaw had trained as a Neurologist in New Zealand before coming to Cambridge, UK on a Wellcome Trust Fellowship and moving to King’s College London in 1995. He is Director of the Maurice Wohl Clinical Neuroscience Institute, and Centre Director of the UK Dementia Research Institute at King’s. His research team have discovered more amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) genes than any other laboratory, enabling gene testing for patients and at-risk family members. They have generated a large number of stem cell and transgenic mouse models that recapitulate key features of the human disease and have revealed important mechanistic insights. Their focus for the future is to develop gene therapies for a wide range of neurodegenerative disorders using adeno-associated viral gene vectors. Award highlights include the “Forbes Norris Award” for Amyotrophic Lateral Sclerosis Care and Research (2009), “Sheila Essey Prize” for Amyotrophic Lateral Sclerosis Research (2012) and “Kea World Class New Zealander Award” (2019). He remains clinically active, running an ALS clinic at King’s College Hospital and leading clinical trials of antisense oligonucleotide therapies.
Seminars
- Re‑examining SOD1 as a therapeutic target in ALS using updated gene‑silencing and delivery approaches informed by recent preclinical work
- Development of a physiological miRNA‑based gene silencing platform using multi‑guide designs to modulate single genes or ALS‑relevant pathways
- Preclinical efficacy and expression data in ALS mouse models, including opportunities for combining gene silencing with protein supplementation
- Engineering a BBB‑crossing capsid to enable durable, pan‑CNS delivery of RNA therapeutics for ALS and related neurodegenerative diseases
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
