Introduction
Gene therapy holds great promise as a potential treatment for Parkinson’s disease, a disorder for which currently available medications do not causally treat the underlying disease mechanisms. However, given the early stage of gene therapy research in this therapeutic area, questions abound regarding its utility and practicality in treating Parkinson’s disease. Those questions have important implications for gene therapy clinical programs, underscoring the importance of early engagement with regulators, payers, and the Parkinson’s patient community.
Since the first gene therapy clinical trial in 1990, nearly 3,000 such trials have been completed or are currently ongoing, 1 a reflection of scientists’ growing appreciation of the transformational potential of gene therapy for a variety of conditions – particularly those with few or no viable treatment options.