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ASHA-623
- Development stage
- Preclinical
- Lead developer
- First Atom Therapeutics
- Modality
- Small Molecules
01
Overview
ASHA-623 is a small molecule mitofusin activator being developed by Asha Therapeutics in collaboration with the Charcot-Marie-Tooth Research Foundation (CMTRF) for the treatment of Charcot-Marie-Tooth disease type 2A (CMT2A). CMT2A is a rare genetic neurological disorder caused by mutations in the MFN2 gene, which encodes the mitofusin 2 protein responsible for mitochondrial fusion and transport. ASHA-623 was designed using Asha's PRISM (Proteostatic Regulation of Intracellular Specific Mechanisms) technology to specifically target and activate mitofusin proteins, thereby restoring mitochondrial fusion and health. The program is currently in the discovery stage of development.
02
Targets
MFN2 (Mitofusin-2)
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