Kelly Glajch
Scientific Director, Rare Neurology- Neuroscience Therapeutic Area Unit Takeda Pharmaceutical
Experienced neuroscientist with 16+ years in industry and academia studying neurological diseases, including Parkinson’s disease, Huntington’s disease, and Amyotrophic Lateral Sclerosis. Experienced as a group leader and directing small and large matrixed teams to advance discovery projects with a range of modalities. Expertise with in vivo models, gene therapy and molecular and cellular neuroscience. My passion is to drive advancement of novel therapeutics to treat neurological diseases.
Seminars
- What is the current understanding of TDP‑43 pathology across ALS, FTD, and related neurodegenerative diseases, and how does it inform therapeutic strategies?
- How can therapies be designed to target upstream TDP‑43 mislocalization, aggregation, or loss-of-function, independent of clinical symptom presentation?
- The role of biomarkers and imaging in detecting subclinical TDP‑43 pathology and enabling earlier intervention in patients
- Challenges and opportunities in translating preclinical findings into human studies, including target engagement, dosing strategies, and safety considerations
- Potential combinatorial approaches, such as pairing TDP‑43-targeted therapies with neuroprotective or gene-modulating modalities to enhance efficacy
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
