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This PRMT6 inhibitor is a small molecule therapeutic candidate being developed by the Agora Open Science Trust in collaboration with M4ND Pharma Inc. for the treatment of neurodegenerative disorders, specifically Spinal Bulbar Muscular Atrophy (SBMA), also known as Kennedy's disease. The drug functions by inhibiting Protein Arginine Methyltransferase 6 (PRMT6), an epigenetic enzyme involved in the methylation of histone H3 at arginine 2 (H3R2) and various non-histone proteins. In the context of SBMA, PRMT6 inhibition is explored as a strategy to mitigate the toxic effects of the mutant polyglutamine-expanded androgen receptor, which is the underlying cause of the disease. The program is currently in the discovery and early lead optimization stages as part of an open-science initiative to develop affordable medicines.
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