Explore the Agenda
8:00 am Morning Check-In:
Served with coffee & light breakfast
Workshop A
9:00 am Deciphering the TDP-43 Driven Genetic Overlap of ALS & FTD Towards NextGeneration Therapies
This workshop will explore the shared genetic and molecular mechanisms underlying Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD), providing insights into converging pathways that may inform novel therapeutic strategies. Participants will examine the latest discoveries in disease causing mutations, modifier genes, and cryptic exon biology, and discuss how these findings are guiding target identification and translational research. The session will also cover approaches to leverage genetic insights for biomarker development, patient stratification, and the design of mechanism-based clinical trials. Attendees will gain a clearer understanding of the genetic intersections between ALS and FTD and the implications for next-generation therapies.
Key Discussion Points
- Genetic overlap between ALS and FTD and how shared pathways contribute to disease progression
- Insights from discovery programs and translational research into novel targets and modifier genes
- Integration of genetic findings into biomarker development and patient stratification for clinical trials
- Opportunities and challenges in leveraging genetic insights to design mechanism-based therapies across ALS and FTD
11:00 am Morning Refreshment & Networking
Workshop B
11:15 am Developing More Meaningful Endpoints through Digital Measures & Patient Reported Outcomes (PROs) to Capture What Matters Most to ALS Patients in Clinical Trials & Drug Design
This interactive workshop will explore how clinical trial endpoints can be designed to reflect what truly matters to people living with ALS. Participants will discuss strategies for integrating patient-reported outcomes, functional measures, digital biomarkers, and quality of-life metrics into trial design. The session will highlight collaborative approaches with patient advocacy groups, lessons from ongoing studies, and regulatory considerations for demonstrating clinical meaningfulness. Attendees will gain practical insights into creating endpoints that enhance trial relevance, patient engagement, and therapeutic impact.
Key Discussion Points
- Defining endpoints that capture functional, cognitive, and quality-of-life outcomes meaningful to patients
- Incorporating patient-reported outcomes, digital tools, and real-world data into trial design
- Regulatory perspectives on patient-centered endpoints and demonstrating clinical relevance
- Collaborative models to co-design endpoints with patients and advocacy groups
1:15 pm Lunch & Networking
Workshop C
2:15 pm 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