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Designing Clinical Trials for Gene Therapy - A Paradigm Shift

  • Broadcast in Science
Pharma Talk Radio

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This session, recorded at the 7th annual Chief Medical Officer Summit and moderated by Dr Martin Childers, Asklepios BioPharmaceutical, discusses designing clinical trials for gene therapy.

Topics covered include:

  • How to utilize adaptive trial design within the specific context of a gene therapy - are there any unique aspects of development conducive to this approach?
  • How to ensure flexibility when pushing for an accelerated pathway?
  • Communicating pros and cons of development options and your recommendation to your CEO
  • De-risk identifying the primary efficacy endpoint without a proof of concept phase
  • How to interpret the current FDA guidance
  • Factors to consider around in-vivo and ex-vivo regulatory guidance
  • How to use the guidance to direct the path of your program
  • Global regulatory considerations and how to overcome them 

Panelists include:

  • Edward Conner, MD, Sangamo Therapeutics
  • Kathleen Reape, MD, Spark Therapeutics

Save the date for the 8th annual Chief Medical Officer Summit on April 6-7, 2020 at the Boston Marriott Cambridge, in Cambridge, MA. Learn more at theconferenceforum.org