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Amyl Therapeutics is developing a monoclonal antibody targeting pathological TAR DNA-binding protein 43 (TDP-43) for the treatment of Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD). This therapeutic candidate is part of the company's Pan-Amyloid Immunotherapy (PAI) platform, which utilizes antibodies designed to recognize and bind to misfolded protein aggregates. The antibody aims to selectively target toxic TDP-43 species to facilitate their clearance and inhibit the cell-to-cell spread of protein pathology, thereby potentially slowing disease progression. As of early 2024, the program is in the preclinical optimization phase.
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