Amydis, a San Diego-based biotechnology company pioneering non-invasive diagnostics for neurodegenerative diseases, has been awarded a $2.5 million Phase 2 grant from the National Institute on Aging (NIA). The funding will accelerate the company’s development of advanced imaging technologies designed to detect amyotrophic lateral sclerosis (ALS) and related neurodegenerative conditions through the eye.

ALS, a progressive neurodegenerative disorder affecting nerve cells in the brain and spinal cord, currently lacks a direct molecular diagnostic test. Diagnosis often relies on excluding other conditions, a process that can take 9 to 12 months, delaying potential therapeutic interventions. Amydis is addressing this challenge by targeting a key molecular hallmark of ALS: the protein TDP-43, which is found in over 97% of ALS cases.

“Amydis’ mission is to provide faster, more precise diagnostic tools for patients,” said Dr. Stella Sarraf, founder and CEO of Amydis. “Currently, there is no test that can directly detect TDP-43 in living patients. Our fluorescent ocular tracer has the potential to fill this critical gap and allow clinicians to identify disease markers during a routine eye examination, preserving precious time for therapeutic intervention.”

Amydis’s approach leverages the eye as a window into the central nervous system. The retina contains cells and structures directly connected to the brain, making it possible to observe early biological changes associated with neurodegeneration. Using small-molecule fluorescent tracers engineered to bind specifically to TDP-43 protein deposits, clinicians may soon be able to visualize molecular biomarkers that are otherwise invisible with standard imaging techniques.

The Phase 2 award builds on Amydis’ earlier Phase 1 NIA grant, which supported preclinical studies showing that the fluorescent tracer could successfully detect TDP-43 in retinal tissue samples collected from deceased patients with ALS, frontotemporal dementia (FTD), and limbic-predominant age-related TDP-43 encephalopathy (LATE). These promising results established the feasibility of the retina as a potential site for diagnosing ALS and related disorders.

“With the Phase 2 funding, we will expand our analysis of retinal tissues to map the distribution of TDP-43 across ALS and other TDP-43-related diseases,” said Dr. Sarraf. “We also plan to integrate artificial intelligence tools to detect disease-specific molecular patterns. This could ultimately help differentiate between related neurodegenerative disorders and guide clinical decision-making in the future.”

Experts in the field are enthusiastic about the potential impact of Amydis’ technology. Dr. Merit Cudkowicz, Executive Director of the Mass General Brigham Neuroscience Institute, commented, “Early detection of TDP-43 in patients with ALS could be transformative. A molecular biomarker test in the eye could not only accelerate diagnosis but also improve clinical trial design, drug targeting, and patient access to promising investigational therapies.”

Amydis’s work represents a significant step toward integrating molecular biomarker detection into routine clinical practice, with the potential to revolutionize how ALS and other neurodegenerative diseases are diagnosed, studied, and treated.