What’s in Store for 2026?
Accelerate ALS & FTD Drug Development with Precision, Insight & Hope Driven Innovation
Uniting 100+ key ALS and FTD stakeholders across pharma, biotech, academia, patient and caregiver communities, key research funding organizations and advocacy groups, the 5th ALS Drug Development Summit is the ultimate platform honing industry R&D for safer, more effect and patient-relevant drugs that serve the community, driving a cure for sporadic and familial ALS.
With 30+ expert speakers and a program spanning early target exploration through to clinical design, biomarker strategy, and regulatory engagement, attendees will gain comprehensive insights into genetically validated targets, the evolving biology of TDP 43, adaptive trial design, and embedding the patient voice throughout development.
Agenda Focus
This year’s agenda delves into ASO mediated cryptic exon correction and sigma 1 receptor modulation to cutting edge approaches in combination therapies, gene silencing platforms, next generation in vitro systems, and emerging discovery engines for ALS and FTD.
Explore breakthroughs in spatial transcriptomics and proteomics, AIenabled biomarker discovery, advanced iPSCderived neuronal and glial models, plasma proteomics for early disease detection, and precision approaches to stratifying fast versus slow progressors. Industry leaders from Eli Lilly, Biogen, Novartis, AbbVie, Takeda, QurAlis, Prilenia Therapeutics, Mitsubishi Tanabe Pharma, Sanofi, ROME Therapeutics, Everything ALS, NIH, Johns Hopkins, and more will share data, perspectives, and lessons learned. Alongside enabling technologies from pioneering model providers, CRO innovators, and digital biomarker platforms, patient advocacy voices help ensure the conversation remains anchored to clinical meaningfulness and real-world impact.
Together, the Summit provides the strategic clarity, scientific momentum, and collaborative energy needed to drive a more hopeful and transformative era in ALS and FTD drug development.
2026 Unmissable Highlights
Advancing Genetically Validated Targets for ALS & FTD
Uncover how the latest genomic breakthroughs are illuminating high value targets and modifier genes across ALS and FTD. Experts from Novartis, QurAlis, and Biogen will decode converging disease mechanisms and outline how these insights are shaping the next wave of precision drug discovery.
Translating Disease Heterogeneity Through Next Generation In Vitro Systems
Explore the newest advances in iPSCderived motor neurons, 3D neuronal cultures, and high content screening that are redefining translational relevance in ALS research. Takeda and leading academic teams will showcase cutting-edge platforms designed to better model patient diversity and therapeutic response.
Transforming Clinical Strategy Through Lessons Learned
Gain clarity from recent trial challenges and successes as experts from Johns Hopkins and the NIH share essential insights on enhancing endpoint sensitivity, refining study design, and reducing regulatory risk. Learn how a more informed approach is accelerating meaningful innovation for people living with ALS.
Centering the Patient Voice in Trial Design & Access
Understand how patient reported outcomes, digital biomarkers, and deep advocacy partnerships are reshaping what “clinical relevance” truly means in ALS. Hear from Everything ALS, CPATH, and Tufts Medical Center on codesigning development frameworks that reflect patient priorities and real-world needs.
Fueling Strategic Investment & Collaboration in ALS
Engage with investors and pharma leaders as they share what drives decision-making in an increasingly competitive ALS and FTD landscape. AbbVie, Trace Neuroscience, and RA Capital provide insight into how early biological signals, biomarkers, and patient centered endpoints are influencing funding and partnership strategy.
Revealing Novel Targets with Spatial Omics & AI
See how spatial transcriptomics, proteomics, and AI powered analytics are uncovering previously hidden pathways in ALS. With contributions from Eli Lilly, Verge Genomics, and AC Immune, discover how these technologies are accelerating precision target discovery and biomarker development.
Divide & Conquer
Track B: Clinical & Regulatory
Explore the Full Event Guide
- 30+ World Class Speakers with Cutting Edge Insights
- 10+ Hours of Face-to-Face Networking Opportunities
- 3 Days of Unmissable Data-Driven Content
- 2 Dedicated Tracks of Content Spanning Discovery to Access for the Whole Team
Attending Companies Include
Explore the Agenda
Dive into 2 tracks, spanning discovery – clinical development and access, 3 preconference day workshops and 30+ expert speakers shaping the future of ALS & FTD
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