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Braegen-04 is a second-generation small molecule inhibitor of asparagine endopeptidase (AEP, also known as legumain or δ-secretase) being developed by Shanghai Braegen Pharmaceuticals (Boruijian Pharmaceutical) for the potential treatment of rare neurodegenerative diseases, including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). AEP is a pH-dependent cysteine protease that cleaves key pathological proteins such as tau, amyloid precursor protein (APP), and TDP-43, promoting their aggregation and neurotoxicity. By selectively inhibiting AEP, Braegen-04 aims to prevent the proteolytic cleavage of these proteins, thereby mitigating neurodegeneration and disease progression. The drug is currently in preclinical development.
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