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glial cell line-derived neurotrophic factor
- Development stage
- Phase 2
- Lead developer
- Amgen
- Modality
- Recombinant Proteins and Enzymes, Gene Therapies
- Administration
- Intraputamenal, Intracerebroventricular
01
Overview
**Glial cell line-derived neurotrophic factor (GDNF)** is a naturally occurring protein that supports the survival, protection, and regeneration of dopaminergic neurons, particularly those in the substantia nigra affected in Parkinson's disease (PD). It acts as a neurotrophic factor, promoting neuronal morphological differentiation and neuroprotection against degeneration. GDNF cannot cross the blood-brain barrier, necessitating direct intraputamenal delivery via recombinant protein infusion (e.g., convection-enhanced delivery) or gene therapy using AAV2 vectors (e.g., AAV2-GDNF or AB-1005) to enable sustained endogenous production in brain cells. Developed initially by Amgen with manufacturing transferred to MedGenesis, it has been investigated in multiple clinical trials for PD and related neurodegenerative disorders, showing safety in Phase 1/2 studies but mixed efficacy in placebo-controlled trials due to delivery challenges; ongoing Phase 2 gene therapy trials aim to address this.
- Other names
- GDNF
02
Targets
NCAM1 (Neural cell adhesion molecule 1)RET (Rearranged during transfection receptor tyrosine kinase)GFRα1 (Glial cell line-derived neurotrophic factor receptor alpha 1)
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