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AMT-140
- Development stage
- Phase 1
- Lead developer
- uniQure
- Modality
- Gene Therapies
- Administration
- Intracerebral
01
Overview
AMT-140 is an investigational **AAV2-based gene therapy** for Parkinson's disease that delivers the human **glial cell line-derived neurotrophic factor** gene directly to the putamen using MRI-guided convection-enhanced infusion. By transducing local cells to express GDNF, the therapy is intended to provide sustained neurotrophic support to degenerating nigrostriatal dopaminergic neurons and thereby improve neuronal survival and function in advanced Parkinson's disease. The program has been associated with academic groups including the University of California at San Francisco, Lund University, and the National Institute of Neurological Disorders and Stroke, and has also been linked to industrial development by uniQure and Brain Neurotherapy Bio. Reported clinical experience indicates Phase 1 evaluation with long-term follow-up showing durable safety and no evidence of cerebral parenchymal toxicity over multiple years.
- Other names
- AAV-GDNF
02
Targets
HTT mRNA (HTT / Huntingtin gene)GFRA1 (Glial cell line-derived neurotrophic factor family receptor alpha 1)GDNF (Glial cell line-derived neurotrophic factor)RET (Rearranged during transfection receptor tyrosine kinase)
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