Designing Clinical Trials for Gene Therapy - A Paradigm Shift

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

Learn more at theconferenceforum.org