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GHB-001

Development stage: Preclinical
Lead developer: GH Research
Modality: RNA Therapeutics → Nucleic Acid Therapeutics

Overview

GHB-001 is a preclinical-stage therapeutic candidate being developed by Gatehouse Bio for the treatment of Idiopathic Pulmonary Fibrosis (IPF). The drug is a small RNA (sRNA) therapeutic identified through the company's AI-driven platform, which analyzes tRNA-derived fragments (tRFs) to discover novel disease-driving mechanisms. GHB-001 is designed to modulate specific sRNA pathways that contribute to the progression of lung fibrosis. As of 2024, the program remains in the preclinical phase of development.

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