
At Target ALS, our mission is to accelerate the discovery and development of effective treatments for ALS by breaking down the barriers that slow progress. Each year, our Annual Meeting brings together the brightest minds in ALS research, from academia, industry, philanthropy, and advocacy, to share insights, challenge assumptions, and push the field forward.
We are proud to announce the launch of our 2025 Annual Meeting report: From Barriers to Breakthroughs: Accelerating the Future of ALS Research for All.
This report offers a comprehensive overview of the groundbreaking science, bold conversations, and collaborative momentum that shaped our Annual Meeting in Boston this past May. It reflects not just where we are today, but where we’re going and how we can get there faster, together.
What’s Inside
- Reflections from Target ALS CEO Manish Raisinghani, M.B.B.S., Ph.D., and Founder and Chairman Dan Doctoroff
- A snapshot of the event by the numbers
- A deep dive into Core Day, our platform for foundational ALS research
- Featured sessions on high-priority drug development programs and biological targets, including:
- A candid discussion of the DNL343 trial by Denali Therapeutics
- New frontiers in RNA biology, splice correction, and TDP-43 pathology
- Advances in understanding and targeting microglia, immune dysfunction, and so-called “junk DNA”
- Highlights from our poster showcase, emphasizing early-career innovation. (Curious? Flip through the posters here).
- Progress toward identifying biomarkers that can improve ALS diagnosis, stratification, and trial design
- Final reflections on the state of the field and the road ahead
Importantly, this year’s report underscores a value we hold central to our mission: transparency. We commend Denali Therapeutics for openly presenting the results of their DNL343 trial, despite the outcome not meeting efficacy endpoints. This kind of scientific integrity helps the entire ALS ecosystem learn, adapt, and evolve.
Why This Matters
ALS is a complex, rapidly progressing disease that demands a coordinated, cross-sector response. Every data point, every failure shared, and every insight gained helps us get closer to a future where ALS is no longer a devastating diagnosis.
Our Annual Meeting report is more than a summary; it’s a roadmap. We hope it will serve as a resource for researchers, funders, policymakers, and advocates alike as we continue to break new ground in ALS research and treatment.
View the photo gallery from the Annual Meeting here.
Stay Connected
To stay informed about our latest funding opportunities, data releases, and research highlights, we invite you to subscribe to the Target ALS newsletter. You’ll receive quarterly updates and occasional alerts about major initiatives shaping the future of ALS research.
Thank you for being part of the movement to accelerate progress, for all people living with ALS, and for the generations to come.