Duchenne UK has awarded £500,000 ($653,000) to biotech company MyoGene Bio to support the development of an innovative gene editing therapy for Duchenne muscular dystrophy (DMD). 

The funding is part of Duchenne UK’s Therapeutic Call, a competitive initiative designed to support innovative research with the potential to deliver new treatments for DMD. 

MyoGene Bio was awarded the financing for its gene editing therapy, MyoDys. The therapy aims to permanently delete a mutation hotspot in the DMD gene, restoring the reading frame and enabling production of a shorter but functional dystrophin protein.  

MyoDys produces a dystrophin protein that mimics a subset of Becker muscular dystrophy patients who experience milder symptoms, with some remaining asymptomatic into their 60s. The therapy offers several advantages over existing genetic medicines, including permanent DNA editing, preservation of natural gene regulation, and restoration of a dystrophin protein three times the size of micro-dystrophin. 

The money will enable MyoGene to optimise muscle targeting, assess durability and explore potential treatment regimens that could improve editing efficiency. It is believed this approach could benefit approximately 50% of patients with DMD.  

Emily Reuben OBE, Co-Founder and Chief Executive of Duchenne UK, said: “This project exemplifies the kind of bold, translational science we aim to fund: science that has the potential to make a real difference to the lives of people living with Duchenne muscular dystrophy. We are excited to see how this promising therapy progresses.” 

“This investment will support essential preclinical work for MyoDys,” added Courtney Young, Co-Founder and Chief Executive Officer of MyoGene.  

“We are grateful for Duchenne UK’s partnership in advancing this cutting-edge genetic therapy closer to clinical trials in DMD patients.” 

Bruno Quinney, Content Team, DDW