Combating immunity to AAV

Ensuring as many patients as possible can benefit from gene therapy

The Duchenne Research Fund has awarded a grant to Dr Barry Byrne and Dr Manuela Corti’s team at the University of Florida, whose vital work focuses on overcoming immunological challenges to ensure all boys and young men can benefit from gene therapy treatments for Duchenne muscular dystrophy.

Why is this research needed?

It is extremely positive news for the Duchenne community that there are currently several gene therapy treatments in clinical trial to combat Duchenne. However, around a third of young people already have a pre-existing immunity to the virus that is used to deliver the gene therapy treatment (known as an adeno-associated virus, or AAV). This means that those patients are unable to participate in current gene therapy trials.

In addition, patients who are eligible to receive the gene therapy will develop immunity to the virus after receiving the first dose, so the challenge of immunity must be overcome in order for them to receive any future doses.

What are the aims of the project?

Dr Byrne and Dr Corti’s work involves developing a strategy to allow dosing in cases of pre-immunity, by modifying the immune system. They aim to establish at what threshhold pre-immunity impacts the safety and efficacy of AAV administration, and to develop a protocol to reduce anti-AAV titers in pre-immune individuals with Duchenne.

They believe this work will have a major impact on the application of gene therapy approaches and provide valuable insight into safe and effective AAV-mediated gene therapy for Duchenne muscular dystrophy.

Dr Barry Byrne is the Associate Chair of Pediatrics and Director of the Powell Center for Rare Disease Research and Therapy. Click here to learn more about his work.

Pre-clinical research

£80,000 contribution

Funded in 2019-20

Florida, USA

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