Boston, MA., Oct. 21, 2024 /PRNewswire/ — Nemdx, Inc. today announced a poster presentation at the 2024 Northeast ALS Annual Conference (NEALS) hosted online on October 21-24. The poster, presented by Vito Levi D’Ancona, president of Nemdx, will feature amyotrophic lateral sclerosis (ALS) research, including findings from testing over 300 samples from multiple biorepositories provided by MGH, NIH/NINDS, Spedali Civili di Brescia, and UK draws.
Dr. Ghazaleh Sadri-Vakili, Director of the NeuroEpigenetics Laboratory at Massachusetts General Hospital and co-author of the poster commented, “The findings presented by Nemdx offer a promising new direction in ALS biomarker research. Their innovative approach to measuring TDP-43 function could significantly impact our ability to diagnose and monitor ALS progression.”
Dr. Avindra Nath, Clinical Director of the National Institute of Neurological Disorders and Stroke (NINDS) at NIH and co-author of the poster, stated, “This research represents a significant step forward in our understanding of ALS pathology. The potential of TDP-43 function as a biomarker could open new avenues for both diagnosis and therapeutic development.”
Dr. Emanuele Buratti, Group Leader at the International Centre for Genetic Engineering and Biotechnology (ICGEB) and co-author of the poster added, “Nemdx’s approach to quantifying TDP-43 function in serum is truly innovative. Their results suggest a new paradigm for understanding ALS at a molecular level, which could have far-reaching implications for patient care.”
Dr. Ian Thrippleton, CTO of Nemdx, explained, “Our engineered synthetic short RNA binders have allowed us to develop assays that can measure both the quantity and function of TDP-43 in in 5µl undiluted serum sample. This technological breakthrough has enabled us to identify functional impairment of TDP-43 in ALS patients, independent of its quantity.”
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Poster Abstract
Amyotrophic lateral sclerosis (ALS) is characterized by the aberrant accumulation of TAR DNA-binding protein (TDP-43), implicating its role as a key player in disease pathology. While TDP-43 dysfunction is well-documented in ALS, its potential as a biomarker remains underexplored. This poster presents findings from a multi-site retrospective study examining serum samples from people living with ALS (PALS) and neurologically unaffected controls. Employing innovative assays utilizing Nemdx-engineered oligonucleotides, we quantified both the amount and function of TDP-43 in serum. Results reveal a significant impairment in TDP-43 function in PALS compared to controls, independent of TDP-43 quantity or demographic factors. Importantly, TDP-43 function demonstrates promise as a discriminative biomarker for ALS, with high sensitivity, specificity, and reproducibility. Our study highlights the potential of TDP-43 function in serum as a novel biomarker for ALS, offering new avenues for diagnosis and therapeutic intervention. This work was supported by ALS-Finding-A-Cure and utilized samples from the NEALS biorepository, NIH/NINDS, Brescia Hospital, and UK draws.
About Nemdx, Inc.
Based in the Boston-area, MA, Nemdx Inc. pioneers a revolutionary approach to manipulating RNA beyond nature’s blueprint. Its synthetic short RNA technology reshapes RNA-protein interactions to advance medical science to shape Therapeutics, Drug Diagnostic Co-Development and Diagnostics. Its innovative binding approach enables a deeper understanding of the biochemical dynamics of proteins in complex heterogeneous diseases, enabling novel clinical products. For more information, nemdx.com
About MGH
Massachusetts General Hospital, founded in 1811, is the original and largest teaching hospital of Harvard Medical School. The MGH Research Institute conducts the largest hospital-based research program in the nation, with an annual research budget of more than $1 billion and comprises more than 8,500 researchers working across more than 30 institutes, centers and departments.
About ICGEB
The International Centre for Genetic Engineering and Biotechnology (ICGEB) is an intergovernmental organization that operates as a center of excellence for research, training, and technology transfer in the fields of biomedicine, crop improvement, environmental protection, and biopharma development.
About MGH – Healey Center
The Sean M. Healey & AMG Center for ALS at Mass General Hospital is on a quest to discover life-saving therapies for all individuals affected by ALS. Launched in November 2018, the Healey Center leverages a global network of scientists, physicians, nurses, caregivers, people with ALS, and families working together to accelerate the pace of ALS therapy discovery and development.