Issues regarding gene therapy products for Parkinson's disease: the development of CERE-120 (AAV-NTN) as one reference point.

PURPOSE: To develop CERE-120 (AAV-NTN) as a novel therapy for Parkinson's disease (PD) that might restore function of degenerating dopamine neurons and prevent further degeneration. SCOPE: A nonclinical program demonstrated that NTN expression can be predictably controlled following CERE-120 administration, provides clear evidence of efficacy in numerous animal models and is safe at dose multiples that far exceed those required for efficacy. Preliminary, open label evidence in PD subjects offers corroborative support for these observations. CONCLUSIONS: CERE-120 may represent an important, novel therapy for PD, though the clinical data require confirmation with additional clinical tests, including an ongoing multi-center, double-blinded controlled trial.