The Phase 2 grant supports AI-driven analysis of retinal tissue to identify disease-specific patterns linked to ALS, FTD, and LATE.
Amydis Inc has received a $2.5 million Phase 2 grant from the National Institute on Aging at the National Institutes of Health (NIH) to advance a non-invasive, eye-based approach for detecting TAR DNA-binding protein 43 (TDP-43), a molecular biomarker associated with amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and Limbic-predominant Age-related TDP-43 Encephalopathy (LATE).
TDP-43 pathology is present in approximately 97% of people diagnosed with ALS, yet no blood test or brain scan currently exists to detect it in living patients. ALS is typically diagnosed through a process of exclusion, often taking months or years due to symptom overlap with other conditions.
To address this gap, the San Diego-based company is developing a fluorescent ocular tracer designed to detect TDP-43 deposits using standard ophthalmic imaging technologies, effectively functioning as a simple eye test that could be administered in routine eye care settings.
“For patients and families facing ALS and related dementias, time is everything,” says Dr Stella Sarraf, founder and CEO of Amydis, in a release. “We believe the eye represents a new frontier in neurodegenerative disease detection. Earlier detection has the potential to change how ALS and related diseases are diagnosed, studied, and ultimately treated.”
Building on Phase 1 Findings
The Phase 2 award follows the successful completion of a Phase 1 NIH grant, during which Amydis demonstrated that its proprietary fluorescent tracer can detect TDP-43 deposits in retinal cadaver tissues from individuals with ALS, FTD, and LATE. That work was supported by Target ALS and conducted in collaboration with Georgetown University, Washington University in St. Louis, Mass General Brigham, Cedars-Sinai, and Banner Health. Results were presented at the Northeastern ALS Consortium conference in November 2025 and the International Symposium on MND/ALS in December 2025, with a manuscript currently being prepared for peer-reviewed journal submission.
The Phase 2 grant will fund continued analysis of retinal cadaver tissues to further characterize and map TDP-43 deposits, with artificial intelligence (AI) applied to identify disease-specific molecular patterns across ALS, FTD, and LATE—with the goal of distinguishing these conditions at a molecular level to support precision medicine applications.
Implications for Clinical Trial Design and Drug Development
Researchers involved in the project say the ability to detect TDP-43 earlier could have significant implications beyond diagnosis, including for clinical trial design and patient recruitment.
“Prodromal detection of TDP-43 in people with ALS would be a step forward for our field,” says Merit Cudkowicz, MD, executive director of the Mass General Brigham Neuroscience Institute and director of the Sean M. Healey & AMG Center for ALS, in a release. “A molecular biomarker test to detect TDP-43 in the eye has the potential to facilitate clinical trial design and drug targeting, as well as accelerate patient enrollment in clinical trials providing earlier access to promising investigational therapies.”
Because patients already visit eye care providers when they notice changes in vision, Amydis says the approach could allow molecular biomarker detection of neurodegenerative disease to be integrated into existing clinical workflows without requiring specialized infrastructure.
