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

Life Sciences, Clinical Trials, Patient Recruitment & Retention, Drug Discovery & Development, Cell and Gene Therapy,
  • Wednesday, April 14, 2021

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.


Andreas Schreiner, Premier Research

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.

Message Presenter

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.

Message Presenter

Who Should Attend?

  • Companies currently in or entering Phase 1 or Phase 2 clinical trials
  • CEO/COO of biotechs
  • Regulatory executives

What You Will Learn

The topics of this webinar will 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

Xtalks Partner

Premier Research

Premier Research, a clinical research company, is dedicated to helping biotech, specialty pharma, and device innovators transform life-changing ideas and breakthrough science into new medical treatments. As a global company, Premier specializes in the use of innovative technologies for smart study design and trial management to deliver clean, conclusive data to sponsors. Whether it’s developing product lifecycle strategies, reducing clinical development cycle times, securing access to patients, navigating global regulations, maximizing the impact of limited rare disease data, or providing expertise in specific therapeutic areas, Premier is committed to helping its customers answer the unmet needs of patients across a broad range of medical conditions. Visit premier-research.com.

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