Sarepta Therapeutics has shared progress on the MyoAAV program and an exclusive licensing agreement with the Broad Institute of MIT and Harvard (Broad Institute) for MyoAAV next-generation capsids for rare genetic diseases like Duchenne muscular dystrophy.
MyoAAV is a new group of adeno-associated viruses (AAV) that use a modified outer protein shell of AAV, known as the capsid, to deliver genetic therapies with greater efficiency and at lower doses. Data published in the journal Cell in 2021 found that, in mouse models of Duchenne muscular dystrophy and X-linked myotubular myopathy, MyoAAV demonstrated more efficient delivery of gene therapy and gene editing payloads, resulting in complete restoration of muscle function and improved survival. In preclinical data from non-human primates, compared to natural AAV serotypes, MyoAAV:
- Delivered 25-50 times greater gene expression in multiple skeletal muscles and 10-15 times greater gene expression in cardiac muscle;
- Demonstrated reduced delivery to the liver by 50 percent and showed lower accumulation in the liver;
- Can be used at up to a log lower dose than traditional AAV vectors due to increased efficiency.
“Research published by Broad Institute, and so far corroborated by Sarepta’s own internal research, reinforces the potential of MyoAAV as a breakthrough next-generation approach in genetic medicine delivery.
The significantly improved efficiency of MyoAAV may unlock the ability to effectively deliver genetic medicine at as much as a log lower doses when compared to current AAVs, which could substantially reduce viral load and cost of goods in the future. As one of the leaders in the use of AAV-mediated genetic medicine to treat rare disease, we intend to push the science forward, and our license for MyoAAV is a quintessential example of that effort.”Doug Ingram, President and Chief Executive Officer of Sarepta
Defeat Duchenne Canada will continue to monitor the progress of this delivery system and share it with our Canadian community.