Neuropeutics is a pharmaceutical company developing targeted therapies to extend neurodegenerative disease patients’ survival and improve their quality of life. One shared pathology across the wide range of neurodegenerative diseases (e.g., Alzheimer’s disease, amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD)) is TDP-43 pathology, which is associated with causing neurotoxicity and neuronal loss.
At Neuropeutics, we have had great success in developing novel high-throughput in vitro and in vivo models of TDP-43 pathology. Using these models, we identified an effective small molecule “JRMS-22” reducing pathology by 30% after two weeks of treatment in a mouse model. Based on our mechanistic understanding of disease, we have developed a novel approach to modulate, prevent, and reverse TDP-43 aggregation. Currently, our team is working relentlessly to finish IND enabling studies in preparation for clinical testing in those living ALS as our initial beachhead market.
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