CERE-135

CERE-135 is an investigational gene therapy developed by Ceregene (acquired by Sangamo Therapeutics) for the treatment of Parkinson's disease. It utilizes an adeno-associated virus serotype 2 (AAV2) vector to deliver the gene for neurturin (NTN), a neurotrophic factor that promotes the survival and functional maintenance of dopaminergic neurons. The therapy is administered via stereotactic neurosurgical injection into the putamen and substantia nigra. The rationale behind CERE-135 was to provide long-term, localized expression of neurturin to halt or reverse the neurodegeneration characteristic of Parkinson's. However, in a randomized, sham-surgery controlled Phase 2 trial, CERE-135 failed to meet its primary endpoint of improving motor function as measured by the Unified Parkinson's Disease Rating Scale (UPDRS). Development was subsequently discontinued for Parkinson's disease, and although it was also explored for Amyotrophic Lateral Sclerosis (ALS), those efforts were also terminated.

Other names

AAV2-neurturinAAV-2-neurturinAAV 2-neurturinneurturin gene therapy