Gene Therapy for Rare Diseases: Considerations for Both Clinical and Post-Marketing Studies, New Webinar Hosted by Xtalk

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There are approximately 7,000 distinct rare diseases affecting 350 million people worldwide, approximately 80 percent of which are caused by faulty genes. Scientific advances such as the CRISPR/Cas9 genome-engineering system have simplified the pharmaceutical and biotech industry’s ability to develop gene therapies, especially for single gene mutation disorders. The US Food and Drug Administration (FDA) has more than 700 active Investigational New Drug Applications (INDs) for gene and cell therapies and in 2017, the FDA approved two cell-based gene therapies and it is anticipated that gene therapy will become a mainstay treatment for many rare diseases.

Xtalks Life Science Webinars

In this webinar, challenges of rare diseases studies focusing on gene therapy treatments, including appropriate study design, statistical analyses and long-term post-authorisation pharmacovigilance and risk management requirements will be examined. Use of appropriate data capture and data quality oversight methods will also be discussed.

Join Karen Ooms, Executive Vice President and Head of Statistics at Quanticate, David Hukin, Managing Director at QVigilance, and Claude Price, Associate Director of Clinical Data Management at Quanticate for a live webinar on Tuesday, January 29, 2019 at 11am EST (4pm GMT) to learn about:

  • Appropriate study design and statistical analyses
  • Long-term post-authorisation pharmacovigilance
  • Risk management activities
  • Appropriate data capture methodologies
  • Key considerations for improving data quality (Data Quality Oversight techniques)

For more information or to register for this event, visit Gene Therapy for Rare Diseases: Considerations for Both Clinical and Post-Marketing Studies


Xtalks, powered by Honeycomb Worldwide Inc., is a leading provider of educational webinars to the global life science, food and medical device community. Every year thousands of industry practitioners (from life science, food and medical device companies, private & academic research institutions, healthcare centers, etc.) turn to Xtalks for access to quality content. Xtalks helps Life Science professionals stay current with industry developments, trends and regulations. Xtalks webinars also provide perspectives on key issues from top industry thought leaders and service providers.

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Candice Tang
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