MyoGene Bio is developing cutting edge therapeutics for muscle diseases, starting with a gene editing therapy for Duchenne muscular dystrophy.
Market: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.
Technology: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
- Methods and compositions for modifying a mutant dystrophin gene in a cell’s genome; WO2017139505A3
- With a deft snip, potential treatment emerges for deadly childhood Duchenne Muscular Dystrophy | LA Times
- Stem cell gene therapy could be key to treating Duchenne muscular dystrophy | UCLA Newsroom
- A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells | Cell Stem Cell
- Creation of a novel humanized dystrophic mouse model of Duchenne muscular dystrophy and application of a CRISPR/Cas9 gene editing therapy | Journal of Neuromuscular Diseases