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Expansion Therapeutics is a biotechnology company focused on the discovery and development of small molecule medicines for RNA-mediated diseases. The company utilizes its proprietary SMiRNA™ platform, based on research from the laboratory of Dr. Matthew Disney, to identify and optimize small molecules that bind to specific, disease-causing RNA structures. Their primary focus is on expansion repeat diseases, such as Myotonic Dystrophy Type 1 (DM1), Amyotrophic Lateral Sclerosis (ALS), and Frontotemporal Dementia (FTD). By targeting the underlying RNA drivers of these conditions, Expansion aims to provide transformative oral therapies for patients with limited treatment options.
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