Pursuing Parkinson’s Disease Gene Therapies: Strategies/Operational Requirements

While treatment of Parkinson’s disease has traditionally been limited to dopamine replacement therapy to alleviate symptoms, gene therapy studies have recently evaluated both non-disease-modifying and disease-modifying transgenes for Parkinson’s disease (PD) treatment, with encouraging results. Non-disease-modifying therapies have improved symptomatology, and disease-modifying strategies – including stopping PD-mediated cell death and/or regenerating lost neurons – show potential to slow disease progression. However, these studies face unique challenges due to their patient population and the complex regulatory considerations for these advanced therapies. Conducting these trials requires careful attention to investigator and site selection and adherence to local regulatory and protocol design requirements. The need for long-term patient follow-up – potentially as long as 15 years – puts added demands on patients and sites. The topics of this webinar include: - Alternate gene therapies such as opto- and chemogenetics and novel genome-editing technology - Use of engineered, non-replicating viral vectors, predominantly recombinant adeno-associated virus or lentivirus - Meeting requirements for surgical techniques and equipment when performing intra-CNS administration of viral vectors - Operational considerations for Parkinson’s gene therapy studies Presenters: Andreas Schreiner, M.D., Vice President, Medical Affairs Neuroscience & Analgesia, Premier Research Hanna Wide, Executive Director, Gene and Cell Therapy, Premier Research