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GHB-003 is a preclinical therapeutic candidate being developed by Gatehouse Bio for the treatment of Alzheimer's disease. The asset was identified using the company's proprietary AI-driven platform, which focuses on small non-coding RNAs (sncRNAs) as both biomarkers and therapeutic targets. Gatehouse Bio's approach involves identifying disease-driving sRNA signatures and developing sRNA-based or sRNA-targeting therapeutics to modulate these pathways. As of early 2024, GHB-003 remains in the preclinical stage, and specific details regarding its molecular target or precise mechanism of action within the Alzheimer's pathology have not been publicly disclosed.
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