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Nepenthe Therapeutics is a pre-clinical stage biotechnology company focused on developing disease-modifying treatments for neurodegenerative diseases, with a primary emphasis on Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD). The company's research is centered on the C9orf72 mutation, which is the most common genetic cause of both ALS and FTD. Nepenthe's approach involves targeting the toxic dipeptide repeat proteins that accumulate in the brains of patients with this mutation. By inhibiting the production or promoting the clearance of these toxic aggregates, the company aims to prevent neuronal death and slow disease progression. Founded as a spin-out from Thomas Jefferson University, Nepenthe leverages the expertise of world-renowned ALS researchers to advance its therapeutic pipeline.
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