Joining Jack has contributed £200,000 as part of £655,000 grant from Duchenne UK to Evox Therapeutics to investigate new ways of delivering gene therapy to patients
This is an exciting time for research into Duchenne Muscular Dystrophy (DMD). Several companies are now testing an approach that uses a synthetic gene to replace the faulty dystrophin gene in Duchenne. This is known as gene transfer using micro-dystrophin. Or more commonly, gene therapy. The companies are using viruses to deliver the therapy.
The early data looks very promising. But there are some challenges in getting this treatment to the entire DMD population, mainly because some patients will have pre-existing antibodies to the virus’ and so will not currently be able to have the treatment.
The £655,000 is being granted to a pre-clinical DMD program with Evox Therapeutics, a company looking at using exosomes to deliver the gene without using a virus.
Exosomes are small nanometer-sized vesicles that occur naturally in cells and carry out different functions. They are essentially small sacs of membrane which carry nucleic acids and proteins. Nucleic acids are large molecules where genetic information is stored, such as DNA and RNA. Think of exosomes like little parcels which deliver information around the body.
Part of Joining Jack's mission is to make sure we find and fund projects that cover all areas of research that demonstrate a potential to treat and cure DMD. We want to leave no stone unturned!
We are really excited to be supporting a project investigating a new potential method of gene therapy delivery. If successful, then we are nearer our ultimate aim of all DMD patients producing their own dystrophin
Dr Antonin de Fougerolles, Chief Executive Officer of Evox, said: “We are excited to be working with Duchenne UK on exploring a potential transformative solution to treat Duchenne patients. We will conduct research to assess the potential of our exosome drug platform to deliver functional dystrophin which is missing or defective in these patients. This work will also allow us to explore targeted delivery of exosomes to muscle which may be beneficial not only for Duchenne patients, but also ultimately for patients with other musculoskeletal diseases.”
Alex Johnson, founder of Joining Jack and co-founder and CEO of Duchenne UK said: “We are delighted to be working with Evox to advance this potentially exciting work to help in the field of DMD. One of the most challenging aspects of using viruses to deliver gene therapy is that many patients may already have what are known as pre-existing antibodies – they are ‘resistant’ to the virus - and so the replacement gene carried by the virus will never reach its target. Exosomes could provide a potential new method for effectively, safely, and repeatedly delivering genetic material encoding for dystrophin to muscles without the problem of pre-existing antibodies.”
We would like to thank our partner charities for their contribution to this project: Alex's Wish - Conquering Duchenne, Caring for Connor Campaign, Chasing Connor's Cure, Duchenne Now, Hope for Gabriel.
We would like to also thank our family funds for supporting the project: Archie’s March, Help Harry fight Duchenne, Hope for Harry, JACK'S MISSION to cure Duchenne Muscular Dystrophy DMD, Jacobi's Wish, Lifting Louis, Project GO, Smile with Shiv, Team Dex, Team Felix and Williams Fund.