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AX-451
- Development stage
- Preclinical
- Lead developer
- Spruce Biosciences
01
Overview
AX-451 is a biological product in preclinical development by Allievex Corp. It is being investigated primarily for the treatment of GM2 gangliosidoses, a group of lysosomal storage disorders that includes Tay-Sachs disease and Sandhoff disease. These are rare, inherited neurodegenerative conditions characterized by progressive destruction of nerve cells due to accumulation of GM2 ganglioside in neurons. The drug's mechanism of action and specific molecular target have not been publicly disclosed as of the latest available information. Its therapeutic focus spans nervous system diseases, congenital disorders, and endocrinology/metabolic diseases[1][2][4].
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