EHC Gene Therapy Guidebook

This Guidebook was put together by the EHC to help support and guide our National Member Organisations (NMOs) through the introduction of gene therapy as a therapeutic option in their countries.

It contains important information, questions, and emerging areas for consideration and food for thought. It also outlines sources of additional information, resources, existing good practices, templates, and recommended actions.

Disclaimer: The EHC produces this publication primarily as an educational tool for our NMOs. The information and views expressed herein constitute the collective input of the EHC Steering Committee, Medical Advisory Group, and international experts in the field of haemophilia.

The EHC does not endorse any particular treatment product or manufacturer; any reference to a therapeutic candidate, product, or treatment class is not an endorsement by the EHC.