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Neurturin gene therapy
- Development stage
- Phase 2
- Lead developer
- Sangamo Therapeutics
- Modality
- Recombinant Proteins and Enzymes, AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Stereotactic Injection, Intrasalivary Gland Injection, Intraocular Injection
01
Overview
CERE-120 is an investigational gene therapy consisting of an adeno-associated virus serotype 2 (AAV2) vector engineered to deliver the human neurturin (NRTN) gene. Once administered—typically via stereotactic injection into specific brain regions—the vector transduces cells to produce neurturin, a neurotrophic factor structurally related to glial cell line-derived neurotrophic factor (GDNF). Neurturin supports the survival and function of dopamine-producing neurons in the substantia nigra and putamen. The primary mechanism is believed to be restoration and protection of degenerating nigrostriatal neurons by providing sustained local expression of neurturin protein. CERE-120 was developed for diseases involving neuronal degeneration such as Parkinson’s disease and has also been investigated for irradiation-induced salivary hypofunction[1][3][6][8].
- Other names
- Neurturin gene therapy
02
Targets
GFRα1 (Glial cell line-derived neurotrophic factor receptor alpha 1)GFRA2 (GDNF family receptor alpha-2)RET (Rearranged during transfection receptor tyrosine kinase)NRTN (Neurturin)
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