MyoGene Bio is developing cutting edge therapeutics for muscle diseases, starting with a gene editing therapy for Duchenne muscular dystrophy.


Duchenne muscular dystrophy (DMD) is a severe, progressive muscle wasting disorder that affects approximately 1 in 3,500-5,000 male births worldwide. It leads to premature death in the 20-30s. There is currently no cure and only limited treatments for DMD. MyoGene Bio's therapy, SPY-DYS45-55, would be applicable to approximately 50% of patients, meaning about 25,000 patients in the US and Europe or 190,000 patients worldwide. The overall dystrophin therapy market is estimated to reach $11B by 2026.


MyoGene Bio's therapy, SPY-DYS45-55, is a gene editing approach designed to permanently remove a mutational hotspot in the gene that causes Duchenne and allow for dystrophin, the protein otherwise lacking, to be expressed. This directly rescues the underlying cause of disease. By targeting a large region of the gene, MyoGene Bio's single therapy is applicable to a large cohort of Duchenne patients and is expected to create a very functional protein that would improve disease progression to a milder form.


  • Permanent gene editing
  • Targets 50% of patients
  • Transforms to mild disease progression
  • Strategies to overcome immune response

Development Stage:

  • Pre-clinical

Patents Issued:

  • Methods and compositions for modifying a mutant dystrophin gene in a cell’s genome; WO2017139505A3

Select Publications:



Graduated in 2022

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