DMD research

Duchenne UK grants $500,000 to overcome immunity challenges in gene therapy

The aim of this project is to give every DMD patient access to gene therapy

6th July 2020

We have partnered with one of the world’s leading gene therapy teams focusing on AAV immunology, Drs. Manuela Corti and Barry Byrne at the Powell Gene Therapy Center, University of Florida to try to temporarily modify the immune system so that anybody can be treated with gene therapy, and, if necessary, can receive repeat doses.

There are currently several gene therapy programmes in the clinic, which all use a virus to try to replace the faulty dystrophin gene. The early data looks promising.

But there are challenges around immunity to the virus used to deliver the gene therapy, that we need to overcome to get this treatment to the entire DMD population,

Challenge 1: Natural immunity

Many people with DMD already have a natural immunity (sometimes described as pre-existing antibodies) to the virus, so their immune system would fight the virus and it would not be able to deliver the replacement gene therapy.

Challenge 2: Repeat dosing

People without natural immunity who can get one dose of gene therapy will develop immunity after their first dosing, so they cannot have more than one treatment.

We are funding this project to find solutions to BOTH the immunity issues.

Drs. Byrne and Corti are developing a newly designed piece of genetic code, to be delivered by the AAV virus. This will be tested in mdx mice using different regimes of immunosuppression devised by the Powell Gene Therapy Center team.

Our main goal is to identify the best strategy to modify the immune systems so that patients can receive gene therapy - both those with pre-existing immunity and those who require repeat dosing.

This is the first stage of a larger development approach. If the team is successful in this first phase, they will go on to test the best code and the best immunosuppression regimen in other DMD animal models more closely related to the human condition.

Drs. Manuela Corti and Barry Byrne at the Powell Gene Therapy Center, University of Florida, said

We are excited to collaborate with Duchenne UK and to take the next step in fulfilling our commitment to patients and families living with DMD. This project is an important milestone in the development of a successful gene therapy treatment that could dramatically improve the quality of life for patients with DMD of all ages to ensure durability and safety.


Emily Crossley and Alex Johnson, co-founders Duchenne UK said:

Duchenne UK is committed to helping where it can to advance the development of gene therapy to and ensure that this promising treatment will be available for all patients regardless of the challenge of antibodies. We are very pleased to be announcing this project and are very grateful to our supporters in helping to fund this.

To find out more about this project, please join our webinar this Wednesday 8th July at 6pm. Our co-founders will be joined by Duchenne UK’s Director of Research Dr. David Bull and Drs. Manuela Corti and Barry Byrne to discuss gene therapy and this new project. 

Register here: https://us02web.zoom.us/webinar/register/WN_mB5HIbDPQ-yR0OcMJ_GzUA


Duchenne UK would like to thank Joining Jack, Duchenne Now and Jacobi’s Wish for generously supporting this project.

We would also like to thank the following partner charities and family funds: Project Go, Cure4George, Smile with Shiv, Team Felix, Following Felix, Team Oscar, Defending William Against Duchenne, Jack's Mission, Chasing Connor's Cure, Help Harry, Action for Arvin, Backing Jack, William's Fund, Jack's Aim, Lifting Louis.

To understand more about gene therapy and the related immunity challenges, please watch this short film:


What is Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease.

Who are Duchenne UK?

Duchenne UK has one clear aim – to end Duchenne. We are the largest funder of Duchenne research in the UK. We connect leading researchers with industry, governing bodies and families to challenge every stage of drug development. Together, we will find treatments and a cure for this generation of patients with Duchenne.

Our president is HRH The Duchess of Cornwall. Our patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

We need your help, because we need to keep funding promising new research.

How to donate?

Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. This can be done via:

  • Direct Debit – Duchenne Direct
  • Individual Donation – Donate
  • If you are a family or friend affected by DMD you can set up your own fund with Duchenne UK – Family and Friend Funds
  • Take part in one of our fundraising events – Events
  • Text DUCHENNE to 70085 to donate £5. This costs £5 plus a std rate msg.

For more information visit www.duchenneuk.org

Published on 7 March 2020

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