Introduction
The Corals-Postdoctoral Fellowship Program supports early-career scientists pursuing research in C9orf72-associated ALS and FTD, providing up to $210,000 over three years.
Katie Copley, PhD, is a postdoctoral researcher at Yale University and a recipient of the Corsalex Postdoctoral Fellowship.
Her work focuses on understanding the role of C9orf72 repeat expansions in ALS and frontotemporal dementia (FTD), with the goal of identifying new therapeutic targets.
In this Q&A, Katie shares her research focus, the challenges of studying ALS, and how the Corsalex Fellowship is helping to advance her work.
Can you tell us a bit about your background and what led you to ALS research?
I was initially drawn to neurodegeneration through personal experience—my grandmother had Alzheimer’s disease. That exposure made me interested in understanding these diseases at a biological level.
During my undergraduate work at the University of Pittsburgh in Chris Donnelly’s lab, I started studying ALS and FTD more directly. That’s when I really saw how devastating these diseases are and how limited treatment options still are. It made me want to stay in this space.
Since then, through graduate school and now as a postdoc in Junjie Guo’s lab at Yale, I’ve continued working on ALS and FTD from different angles.
What made the fellowship a good fit for your work?
Corsalex has a strong focus on C9orf72-related ALS and FTD, which aligns directly with my research. My work centers on the C9 repeat expansion, so it felt like a very natural fit between my project and their priorities.
Can you describe your research focus?
My research focuses on C9orf72 ALS and FTD, the most common genetic cause of both diseases.
In this form of ALS, a section of DNA in the C9orf72 gene contains a repeat sequence. In healthy individuals, there are typically fewer than ~20 repeats. In disease, this can expand to hundreds or even thousands.
I’m studying how this expansion changes over time and how the length of the repeat may influence disease biology and clinical outcomes.
What is the central question your work is trying to answer?
The main question is whether the length of the DNA repeat expansion itself has a direct effect on toxicity and disease phenotype.
There’s been a lot of work on the RNA and protein products derived from the repeat, but less focus on the role of the DNA expansion itself. Emerging evidence suggests the DNA may contribute to disease in its own way, and I’m trying to understand how changes in repeat length might influence disease—and whether that could be modulated.
Why is this important for developing treatments?
If the DNA repeat itself is contributing to disease, that represents a very upstream mechanism.
We know the mutation is causative, but not exactly how it drives pathology. If we can directly target the expansion—or its effects—we may be able to intervene closer to the root cause of disease rather than downstream consequences.
What are the biggest challenges in this area of research?
There are two main challenges.
First, the biology is complex. There’s a large body of literature across many different mechanisms, and part of the challenge is integrating those insights to identify where interventions might be most effective.
Second, the repeat expansion itself is technically difficult to study. It’s highly GC-rich, which makes sequencing and characterization challenging. Even obtaining accurate measurements can be a hurdle, so both the technical and conceptual aspects require careful approach.
What approaches or data sources are you using to study this?
I’m drawing on several areas.
There’s increasing interest in DNA damage pathways in C9 ALS/FTD, which may be directly relevant to the repeat expansion. I’m also looking at insights from other repeat expansion disorders, like Huntington’s disease, where modulation of repeat length has been studied more extensively.
Even though these are different diseases, some of the underlying principles may translate. A big part of the work is integrating knowledge across fields to apply the best possible approaches.
What do you hope to achieve through the fellowship?
One of the key goals is to identify ways to modulate the C9 repeat expansion.
If we can move toward that, it could point to a strategy that’s potentially translatable. Ultimately, the goal is always to understand how this work could contribute to developing treatments for patients.
How does your work connect to the drug development process?
Right now, my work is focused on understanding the biology of the repeat expansion.
The next step would be identifying genetic modulators—factors that influence the expansion. If we can identify one, that becomes a potential therapeutic target.
From there, there are multiple ways to pursue it—small molecules, AAV-based approaches, ASOs. The key is identifying a target that can be developed using approaches that are already being applied in other diseases.
How has the Corsalex Fellowship supported your work?
It’s been a really valuable complement to the academic environment.
At Yale, I have access to strong academic resources and collaborations. Through Corsalex, I’ve been able to engage with a team that has experience in translating research toward clinical development.
They’ve been very supportive of the project, and it’s been helpful to get feedback from people with different perspectives. It also provides insight into how discoveries might move into a development pipeline.
What role does Corsalex play more broadly in ALS research?
Corsalex is focused on advancing therapies for C9orf72 ALS and FTD.
They’re working to move from target discovery into clinical development, while also building internal research capabilities to better understand disease mechanisms and identify new targets. They’re also actively engaging with academic and industry partners to accelerate progress.
What’s your perspective on where ALS research stands today?
It feels like a promising moment.
There are still many unknowns, but there has been real progress—particularly with the approval of therapies like tofersen for SOD1 ALS. That’s important because it demonstrates that it’s possible to develop treatments that target the underlying biology.
Even though that applies to a specific genetic form, it gives confidence that with deeper understanding, similar progress can be made in other forms of ALS.
What excites you most about this field right now?
There have been recent technological advances that make it easier to study repeat expansions, which were previously very difficult to characterize.
That opens up new opportunities to understand disease mechanisms in ways that weren’t possible before. It feels like we’re now in a position to answer questions that have been out of reach.
What keeps you motivated in this work?
The ALS and FTD research community is incredibly motivating.
There’s strong engagement not just from scientists, but also from patients, families, and organizations. Seeing that level of commitment—and how invested people are in advancing treatments—has been a big reason I’ve stayed in this field.
What are your long-term goals?
I plan to continue working in ALS research and ultimately lead my own lab focused on ALS and FTD.
About the Corsalex Postdoctoral Fellowship
Corsalex is a nonprofit organization focused on developing treatments for C9orf72-associated ALS and FTD. Its mission is to identify, fund, and advance promising therapeutic approaches targeting this genetic form of disease, the most common cause of familial ALS/FTD. The organization works with partners across academia and industry, including the ALS Therapy Development Institute (ALS TDI), to help accelerate progress.
The Corsalex Postdoctoral Fellowship Program supports early-career scientists pursuing research in this area, providing up to $210,000 over three years. Applications for the 2026 cycle open May 1 and close July 1, with awards announced by October 1.
For more information, visit https://www.corsalex.com/corsalex-fellowship.