CERE-130

CERE-130 is an investigational gene therapy designed to treat Parkinson's disease by delivering the gene for neurturin (NTN), a member of the glial cell line-derived neurotrophic factor (GDNF) family. Developed by Ceregene (later acquired by Sangamo Therapeutics), the therapy utilizes an adeno-associated virus type 2 (AAV2) vector to achieve long-term expression of neurturin in the brain. The treatment is administered via stereotactic intraparenchymal injection into the putamen and, in some clinical protocols, the substantia nigra. Neurturin is intended to provide neurotrophic support to degenerating dopaminergic neurons, potentially improving their survival and function. Although early Phase 1 trials demonstrated safety and suggested potential efficacy, two subsequent Phase 2 double-blind, sham-controlled trials failed to meet their primary clinical endpoints, leading to the cessation of the program's development for Parkinson's disease.

Other names

AAV2-neurturinAAV-2-neurturinAAV 2-neurturinAAV2-NTNAAV-2-NTNAAV 2-NTN