Drug intelligence / Profile preview

gambogic amide

Development stage
Preclinical
Lead developer
Emory University
Modality
Small Molecules
Administration
Intraperitoneal, Subcutaneous
01

Overview

Gambogic amide is a synthetic small-molecule agonist of the high-affinity nerve growth factor receptor (TrkA). Discovered through a cell-based chemical genetic screen at Emory University, it acts as a functional mimetic of nerve growth factor (NGF) by binding to the cytoplasmic juxtamembrane domain of TrkA, triggering receptor dimerization, tyrosine phosphorylation, and activation of downstream Akt and MAPK signaling pathways. Unlike NGF, which is a polypeptide with poor pharmacokinetic properties, gambogic amide is a stable small molecule that has demonstrated neuroprotective, neuroplastic, and regenerative effects in preclinical models of stroke, neurodegenerative diseases, tendon injury, and bone formation. It is also widely utilized as a selective research tool to study TrkA-mediated signaling.

Other names
GamAmgambogic acid amide
02

Targets

NTRK1 (Tropomyosin-related receptor kinase A)

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