Drug intelligence / Profile preview

HT-VA GDNF

Development stage
Preclinical
Lead developer
Hoth Therapeutics
Modality
Recombinant Proteins and Enzymes
Administration
Parenteral
01

Overview

HT-VA GDNF is a preclinical-stage biologic therapeutic being developed by Hoth Therapeutics in collaboration with the U.S. Department of Veterans Affairs (VA) and Emory University. The drug is a recombinant form of Glial Cell-Derived Neurotrophic Factor (GDNF), a protein traditionally recognized for its neuroprotective properties that has recently been identified as a potent regulator of metabolic gene pathways. HT-VA GDNF functions by reprogramming liver fat metabolism at the genetic level, specifically through the downregulation of the lipogenic gene *Srebf1* and the upregulation of the fatty acid oxidation gene *Pparα*. In preclinical head-to-head studies, the drug demonstrated superior efficacy compared to semaglutide in weight stabilization, glucose tolerance, and the reduction of liver weight and adipose tissue. Hoth Therapeutics plans to advance the program through IND-enabling studies with the goal of initiating clinical trials in 2027 for the treatment of obesity and metabolic-associated fatty liver disease (MAFLD/MASLD).

Other names
Glial Cell-Derived Neurotrophic FactorGDNF
02

Targets

NCAM1 (Neural cell adhesion molecule 1)RET (Rearranged during transfection receptor tyrosine kinase)GFRA2 (GDNF family receptor alpha-2)GFRα1 (Glial cell line-derived neurotrophic factor receptor alpha 1)

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