GHB-005

GHB-005 is a preclinical-stage small RNA (sRNA) therapeutic candidate being developed by Gatehouse Bio for the treatment of Huntington's disease. The drug was discovered using the company's proprietary AI-driven platform, which identifies disease-specific sRNA signatures—naturally occurring small RNA molecules that regulate gene expression and are often dysregulated in pathological states. GHB-005 is designed to modulate these signatures to restore cellular homeostasis in Huntington's disease patients. As of early 2024, the program remains in the preclinical stage of development, with the company leveraging its sRNA platform to address the underlying genetic and molecular drivers of neurodegeneration.