Amydis, a clinical-stage biotech developing imaging tracers to detect disease biomarkers in the eye, has secured a $2.5 million Phase 2 grant from the National Institute on Aging at the National Institutes of Health (NIH) to advance a potential breakthrough in diagnosing devastating neurodegenerative diseases.

The funding will support Amydis’ effort to solve one of medicine’s biggest diagnostic gaps: detecting TAR DNA-binding protein 43 (TDP-43)—a molecular hallmark linked to conditions including amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and Limbic-predominant Age-related TDP-43 Encephalopathy (LATE).

The need is urgent. About 97% of people diagnosed with ALS show TDP-43 pathology, yet there is still no blood test or brain scan capable of identifying the protein in living patients. As a result, ALS is typically diagnosed only after ruling out other diseases—a process that can take months or even years.

Amydis believes the answer may lie in the eye.

The company is developing a non-invasive fluorescent ocular tracer designed to detect TDP-43 using routine ophthalmic imaging technology. The approach could transform a standard eye exam into a screening tool for neurodegenerative disease—potentially enabling earlier diagnosis and faster access to clinical trials and emerging therapies.

"For patients and families facing ALS and related dementias, time is everything," said Stella Sarraf, Founder and CEO of Amydis. "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."

The Phase 2 award follows promising results from a previous Phase 1 NIH grant. In that study, Amydis demonstrated that its proprietary fluorescent tracer could detect TDP-43 deposits in retinal tissue from ALS, FTD, and LATE donors.

The research was carried out in collaboration with leading academic and medical institutions including Georgetown University, Washington University in St. Louis, Mass General Brigham, Cedars-Sinai and Banner Health, with additional support from Target ALS. Findings were presented at the NEALS conference in November and the International Symposium on MND/ALS in December, with a peer-reviewed publication now in preparation.

The new grant will fund further analysis of retinal tissue to map TDP-43 deposits in greater detail. Researchers will also apply artificial intelligence to identify disease-specific molecular patterns across ALS, FTD, and LATE—work that could help distinguish the conditions and support more precise treatment strategies.

"Prodromal detection of TDP-43 in people with ALS would be a step forward for our field," said Dr. Merit Cudkowicz, Executive Director of the Mass General Brigham Neuroscience Institute and Director of the Sean M Healey & AMG Center for ALS.

"A molecular biomarker test to detect TDP43 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."