Technology used in COVID-19 vaccine to be used for Duchenne gene therapy research

Alex’s Wish has funded £30,000 as part of Duchenne UK’s £287,500 investment into a new 18-month study investigating whether Lipid Nanoparticles (LNPs) could be used to help in gene therapy treatments for DMD.  

Lipids are naturally occurring small fatty molecules that exist within the body, and nanoparticles refers to their small size. LNPs are currently being used as a key component in the Pfizer/BioNTech and Moderna COVID-19 vaccine. 

We are now funding work to see whether these LNPs could be used to deliver gene therapy in DMD.  

Several clinical trials of gene therapy are now underway in DMD using harmless viruses, called AAVs, to deliver synthetic gene to replace the faulty dystrophin gene in Duchenne. The early data looks 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. 

There are also difficulties in transferring the genes from the viruses into muscle cells. 

This study aims to address some of these challenges by exploring LNPs as a method of delivering gene therapy. The study is called ‘mRNA Targeted Therapeutics for Duchenne Muscular Dystrophy’ and will be led by Professor Shenhav Cohen at Technion Institute of Technology, Israel, in partnership with Professor Aartsma-Rus at Leiden University Medical Center and Professor Peer at Tel Aviv University.  

LNPs can be filled with a variety of materials, including genetic material. Because LNPs are made of naturally occurring lipids, they should not cause an immune response. They also have a good safety profile for use in humans. 

This study will explore whether a larger dystrophin construct can be carried by the LNPs. The dystrophin gene is the largest gene in the body, but in current gene therapy trials, a shortened construct must be used. Our current understanding of the impact of shortened dystrophin on long-term muscle function is limited, but using a longer gene could lead to better muscle function. The research group plans to engineer the LNP surface such that the LNP will effectively fuse with muscle and deliver its cargo.  

This research is currently in very early stages. If the researchers can show that this method can effectively deliver genetic material into muscles using a mouse model of DMD, they would have a high chance of receiving a much larger grant for further research.   

We are excited to be funding this early research into a safer and more effective way to deliver gene therapy, alongside other projects looking to reduce the immunological issues of using viruses. If successful, this project could have a transformative impact on the way gene therapy is delivered for DMD in future.  

This Project was funded by Duchenne UK with the help of ourselves and Little Steps, and the following Family Funds: Chasing Connor’s Cure, Jack’s Mission, Help Harry, Archie’s March, Muscles for Mitchell, Jacobi’s Wish, William’s Fund, Project Go, Action for Arvin, Following Felix, Defending William Against DMD, Henry’s Hurdles; Ben vs Duchenne.

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