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.

Speakers

Andreas Schreiner, Vice President, Medical Affairs Neuroscience and Analgesia, Premier Research

Dr. Schreiner has considerable experience as an investigator, as well as global experience within the pharmaceutical industry and clinical research environment. In his role, he is responsible for providing strategic planning, coordination, knowledge and expertise in neuroscience and analgesia for projects and/or stand-alone consultative services.

Dr. Schreiner is a certified psychiatrist and neurologist. He studied medicine at the Universities of Heidelberg and Mannheim, Germany, and completed residencies in the University Department of Neurology and in Psychiatry at the Central Institute of Mental Health, both in Mannheim, Germany.

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Hanna Wide, Executive Director, Gene and Cell Therapy, Premier Research, UK

Hanna Wide provides oversight for clinical projects and programs within the rare disease and pediatric space, specializing in gene therapy in rare indications. Her role is focused on operational strategy and oversight of trials including rare genetic, metabolic and rare neuroscience indications.

She has previously worked as a Project Director and Project Manager at Premier Research, managing clinical trials within rare genetic diseases and pediatric research. Ms. Wide has over 10 years of experience in CRO project management in various CROs, most of which has been dedicated to rare disease trials.

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