Duchenne UK awards $500,000 to accelerate development of gene editing therapy for Duchenne muscular dystrophy
We are proud to announce $500,000 in funding for MyoGene Bio, a San Diego-based biotech company, to support the development of an innovative gene editing therapy for Duchenne muscular dystrophy (DMD).
Therapeutic Call
The funding is the result of our Therapeutic Call, a competitive initiative aimed at advancing innovative treatments for DMD.
The project, titled ‘Optimization of a gene editing therapy for Duchenne muscular dystrophy’, will focus on refining MyoGene’s cutting-edge CRISPR/Cas9-based therapy, MyoDys45-55, which aims to permanently delete a mutation hotspot in the DMD gene. This approach is designed to restore the reading frame and produce a shorter but functional dystrophin protein, similar to that found in individuals with Becker muscular dystrophy.
Potential reach
If successful, the therapy could be suitable for approximately 50% of boys with DMD.
The money will enable MyoGene to optimise muscle targeting, assess durability and explore potential treatment regimens that could improve editing efficiency.
This work is expected to lead to MyoDys45-55 being brought to clinical trials within two years.
MyoGene Bio is a biotech startup dedicated to developing transformative therapies for muscle diseases. The Duchenne UK money forms part of a total $1.42 million investment in the program.
“We are grateful for Duchenne UK's partnership in advancing this cutting-edge genetic therapy closer to clinical trials in DMD patients. This investment will support essential preclinical work for MyoDys45-55.”
Courtney Young, PhD, Co-founder and Chief Executive Officer of MyoGene
“We are pleased to support MyoGene’s pioneering work through our Therapeutic Call. 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 DMD. We are excited to see how this promising therapy progresses.”
Emily Reuben OBE, Co-founder and Chief Executive of Duchenne UK