CureC9 Program
A strategic effort to cure the most common genetic cause of ALS and FTD: the C9orf72 repeat expansion.
CureC9 is a research program within EverythingALS, a 501(c)(3), dedicated to curing C9orf72 disease — the most common genetic cause of ALS and FTD. An independent Scientific Advisory Board comprised of eight international experts identifies which research is most likely to change outcomes and directs funding accordingly. One hundred percent of donations go to the science.
Why CureC9 Exists
The C9orf72 repeat expansion is the most common genetic cause of both ALS and frontotemporal dementia. Despite major investment in ALS research, there has not been a program dedicated to strategically directing research specifically for C9 disease.
CureC9 was created to fill that gap.
Through an independent Scientific Advisory Board of therapy-focused scientists, CureC9 evaluates the research landscape, challenges inherited assumptions, and helps direct funding and scientific strategy toward the work most likely to lead to meaningful progress.
Two Arms, One Mission
Prevention Arm
Detecting the disease before symptoms start, so we can intervene while there is still time.
Mechanism Arm
Driving the key science forward to meaningfully alter C9orf72 disease.
Scientific Advisory Board
Cure C9 is guided by an international advisory board of leading experts in ALS, neurodegeneration, and therapeutic development. These scientists help identify the most promising research directions and ensure that funding is directed toward projects with the highest potential to change outcomes for patients.
Claire Clelland (MD, PhD)
John Douglas French Alzheimer’s Foundation Founding Associates Endowed Professor | University of California San Francisco | Weill Institute for Neurosciences
Steven Boeynaems, (PhD)
Assistant Professor | Baylor College of Medicine | Therapeutic Innovation Center (THINC) / Texas Children’s Hospital | Jan and Dan Duncan Neurological Research Institute
Leadership
Yentli Soto Albrecht
MD-PhD student | University of Pennsylvania | C9 Carrier and Co-Founder of CureC9
Ed Rapp (BSBA)
Chair of Answer ALS Advisory Board |Member of Packard Center Board and Executive Committee
Research Priority Areas
The Mechanism Arm is currently organized around four strategic priority areas.
Biomarkers of Disease Divergence
Why does the same mutation lead to ALS in some carriers and FTD in others? This work focuses on identifying the biological signals that may help answer that question.
Failed Trial Analysis
CureC9 seeks to learn from past failed trials so future therapies are built on stronger scientific ground, better biomarkers, and better assumptions.
Closed-Loop Experimental Systems
This area supports systems that test whether therapies improve nerve function and disease biology, not just whether they change a laboratory marker.
Gene Therapy Optimization
This work focuses on improving gene therapy strategies for C9 disease, including delivery systems, molecular design, and approaches aimed at a true molecular cure.
Scientific Advisory Board
CureC9’s Scientific Advisory Board brings together scientists working across genetics, gene therapy, neuropathology, immunology, antisense therapeutics, transcriptomics, and neurodegeneration. Together, they help identify the questions, projects, and strategies most likely to move the field forward.
Help Accelerate the Search for a Cure
CureC9 depends on the work of scientists, supporters, collaborators, and advocates who believe genetic ALS deserves focused, strategic attention. Whether you want to support research, offer your skills, or help spread the word, there are many ways to get involved.
Together, We Can Move This Faster.
This is what You Can Do
Learn From Failed Trials: Turn “No Results” Into New Targets
Goal: $500,000
Why it matters: Trials that didn’t work can still contain the most valuable clues—if we can analyze the right biosamples, we can identify signals that point to why the trial failed and what to try next.
Unlock Gene Therapy Delivery to the Brain
Help us Raise: $10,000,000
Why it matters: If we can reliably deliver gene therapy into the brain, it accelerates not only C9 ALS work, but also broader neurodegenerative diseases (and potentially other brain conditions).