Practical tips for setting up gene therapy trials – an ‘Institutional Readiness’ framework

Enrolment will be open from 9th March 2022


Course Edition: 1st Edition

Starting date of course: 9th March 2022

Ending date of course: 30th April 2024

Starting date of enrolment: 9th March 2022

Ending date of enrolment: 30th April 2024

Course duration: 2.5 hours

General Description

The course gives an overview of some key features of gene therapy clinical trials and how the set-up of these trials can differ from other clinical trials.

The course describes the development of two Institutional Readiness surveys that were designed to investigate how prepared UK sites were to carry out gene therapy trials in the context of the rare neuromuscular disease Duchenne Muscular Dystrophy, and provides a framework for conducting an Institutional Readiness evaluation at participants’ own sites.

As a result of the course, participants will be able to understand key elements that must be addressed when setting up a gene therapy trial and also recognise the importance of a thorough evaluation of Institutional Readiness and be able to adapt key elements of an Institutional Readiness assessment for use at their own sites.



  1. Introduction
  2. Overview
  3. Institutional Readiness Survey #1
  4. Institutional Readiness Survey #2
  5. Learning from experience and resources
  6. Metabolic field Perspective
  7. Industry Perspective

Please click here for course flyer.

Who is the course for?

Members of staff involved in setting up and delivering clinical trials at sites that are interested in gene therapy, including but not limited to, Principal Investigators, Clinical Trial Coordinators and Research Nurses.


Max. number of persons that each national hub can enrol: unrestricted


Bloom’s Taxonomy Level of the Course: 3










Anna Irvin –