Drug intelligence / Profile preview

neurturin

Development stage
Unknown
Lead developer
Sangamo Therapeutics
Modality
Recombinant Proteins and Enzymes
Administration
Parenteral, Gene Therapy (intracerebral), Experimental Subcutaneous (animal Models)
01

Overview

**Neurturin** is a protein growth factor belonging to the glial cell line-derived neurotrophic factor (GDNF) family, classified as a neurotrophic factor and part of the TGF-beta superfamily. It plays a critical role in supporting the survival, development, and maintenance of neurons, particularly midbrain dopaminergic neurons relevant to diseases such as Parkinson's. Neurturin mediates its effects primarily through the GDNF family receptor alpha-2 (GFRα2) and the receptor tyrosine kinase RET, leading to cellular survival signaling and upregulation of tyrosine hydroxylase in the nigrostriatal system. As a protein itself, neurturin has served as a therapeutic transgene in gene therapy clinical trials for neuroprotection and disease-modification in Parkinson's disease, most notably delivered via vectors such as AAV2-neurturin (CERE-120)[1][5][6][3][4][2].

Other names
neurturinNRTNNTN
02

Targets

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

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