What You Missed In 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 was 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 gained comprehensive insights into genetically validated targets, the evolving biology of TDP 43, adaptive trial design, and embedding the patient voice throughout development.
Key Themes That Shaped the 2026 Agenda
This 2026 agenda delved 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.
Attendee's explored 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 shared data, perspectives, and lessons learned. Alongside enabling technologies from pioneering model providers, CRO innovators, and digital biomarker platforms, patient advocacy voices helped ensure the conversation remains anchored to clinical meaningfulness and real-world impact.
Together, the Summit provided 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
We uncovered how the latest genomic breakthroughs are illuminating high value targets and modifier genes across ALS and FTD. Experts from Novartis, QurAlis, and Biogen decoded converging disease mechanisms and outlined how these insights are shaping the next wave of precision drug discovery.
Translating Disease Heterogeneity Through Next Generation In Vitro Systems
Explored 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 showcased cutting-edge platforms designed to better model patient diversity and therapeutic response.
Transforming Clinical Strategy Through Lessons Learned
Gained clarity from recent trial challenges and successes as experts from Johns Hopkins and the NIH shared essential insights on enhancing endpoint sensitivity, refining study design, and reducing regulatory risk. Learnt how a more informed approach is accelerating meaningful innovation for people living with ALS.
Centering the Patient Voice in Trial Design & Access
Understood how patient reported outcomes, digital biomarkers, and deep advocacy partnerships are reshaping what “clinical relevance” truly means in ALS. We heard 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
Engaged with investors and pharma leaders as they shared what drives decision-making in an increasingly competitive ALS and FTD landscape. AbbVie, Trace Neuroscience, and RA Capital provided insight into how early biological signals, biomarkers, and patient centered endpoints are influencing funding and partnership strategy.
Revealing Novel Targets with Spatial Omics & AI
Saw 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, we discovered how these technologies are accelerating precision target discovery and biomarker development.
Divide & Conquer
Explore the 2026 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 Included
Explore the 2026 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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