Management of safety issues arising following AAV gene therapy


Number 261
Date 19 March 2021

Location: Virtual Zoom Meeting

Title: Management of safety issues arising following AAV gene therapy

Date: 19 March 2021

Organisers: Prof. F. Muntoni, Prof. C. Bonnemann, Prof. L. Servais

Participants: Adjali Oumeya, Mimoun Azzous, Diana Bharucha, Michael Binks, Anna Buj-Bello, Barry Byrne, Manuela Corti, Fadi Fakhouri, Andreas Hartmann, Rebecca Horton, Alex Johnson, Caroline Le Guiner, Federico Mingozzi, Carl Morris, Victoria Palmi, Dimah Saade, Sam Hopkins, Andreas Ziegler, Sherif Gabriel

The European Neuromuscular Centre hosts regular workshops in order to enhance collaboration within the neuromuscular disease field. The 261st workshop, organised by Professors Muntoni, Bonnemann and Servais, covers the safety management of AAV gene therapy. The full workshop, which was originally scheduled to take place between the 19th and 21st of March 2021, has been delayed to the 29th to 31st of October in light of the current pandemic. A short introduction meeting took place via zoom videoconferencing on Friday 19th March 2021.

The overall aim of the meeting is to gather together the stakeholders for transparent sharing of information regarding serious adverse reactions observed after gene therapy administration, in order to work out the best strategy to decrease risk and manage these adverse reactions when they occur.

Participants include experts in the field including pharmaceutical companies, regulators, clinicians, academics and patient advocacy representatives. There were participants from multiple specialities in order to gather the expert opinion on the types and mechanisms of adverse reaction. The group included immunologists, nephrologists and haematologists as well as neurologists.

A review of all of the publicly available information on safety and immunology concerns has taken place and been compiled, and initial results have been shared with participants. A complete report is going to be submitted for publication soon. A framework for information sharing among participants was outlined and smaller work groups were created to outline strategies for monitoring, prophylaxis and risk-mitigation of treatment related adverse events in AAV mediated gene therapy in preparation for the meeting in October.

We hope that this collaboration will make AAV Gene Therapies safer for all patients and de-risk the use of promising therapies for clinicians, investigators and partners in academia and industry.