Presented at 2020’s Gene Therapy in Rare Blood Disease event in Boston, MA. Hemophilia is an important sector within biotech, and one of the largest rare disease markets. Design of clinical trials with consideration for the recent regulatory landscape is central for cell and gene therapy biotech firms in this space. This talk focused on:


Attendees learned about:

  • Gaining Insights into a Successful Hemophilia Gene Therapy Trial
  • Overviewing standard outcomes measures and recent surrogate endpoints,
    safety measures, and patient important outcomes (PIOs) and patient reported
    outcomes (PROs)
  • Understanding logistical considerations for early trial clinical development and
    scalable clinical product management
  • Examining patient selection and retention in hemophilia advanced therapy trials


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Preferred citation:

Johnson, A.N. [Workshop] Strategic Gene Therapy Product Platforms and Partnerships: From Codevelopment to Companion Products. Gene Therapy for Rare Blood Disorders March 3-5, 2020. Boston, MA USA. Accessed online at


You can download the slides from this presentation here.