AAV Gene Therapy in the Clinical Setting: A Practical Approach

Scientist in a laboratory

7 Sep 2021 9 a.m. to 7 Sep 2021 12:15 p.m.

Event recording

This event was originally broadcasted on the 7 September 2021

On Demand Webpage: Videos from the individual sessions of the event, with the exception of the round table discussion, are available and can be found here: https://aav-genetherapy.com/on-demand/

About this event

Adeno-associated viral (AAV) gene therapy is very rapidly progressing. Many AAV therapies are being tested in early and more advanced clinical trials, with some recently receiving European Medicines Agency (EMA) approval and becoming available under the NHS. In view of the progress in many of the ongoing clinical trials, it is anticipated that many more AAV gene therapy products will become available for patients currently receiving other, or no, therapies within the NHS.

The handling, administration, and clinical surveillance of patients receiving AAV gene therapy is different to other therapeutic interventions, especially regarding the potential for adverse events and how to best manage those. Several UK hospitals and Biomedical Research Centres already have a wealth of knowledge and experience in running AAV gene therapy trials in several indications. But the availability of AAV gene therapy products for larger numbers of paediatric and adult patients with genetic conditions means it is necessary to build expertise on AAV in many other sites.

This virtual meeting has been set up to address the unique challenges of AAV gene therapy, and provide practical suggestions on how these therapeutic interventions can best be delivered.

Which topics will be discussed?

  • Mechanism of action of AAV gene therapy, and how this differs from other forms of gene therapy
  • How to set up a site for AAV gene therapy: from pharmacy, to Genetically Modified Organisms Safety Committee, to therapy delivery location
  • Preparing patients ahead of the AAV gene therapy
  • Post-discharge planning: safety monitoring and hub-and-spoke models of follow-up
  • Healthcare professionals’ experience and patient and public involvement

Who should attend this meeting

The target audience is primarily those in the delivery team in hospitals who are currently not involved in AAV gene therapy. This includes nurses, coordinators, pharmacists, physicians and other members of the MDT.

Registration and details of the programme

To register for the event and for further details of the speakers and the programme, please visit the following webpage: