What is Gene Therapy?
A gene therapy is a therapeutic approach that uses genes to facilitate production of a properly functioning protein or enzyme to treat or prevent a particular disease. At M6PT, we add a new gene into the body to help fight disease.
There are other approaches using genes called gene editing:
- Replacing a gene that is not working properly and causes disease with a healthy copy of the gene
- Stop or “knocking out,” a gene that is not functioning properly
Although gene therapy is a promising treatment modality for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections) and there are a few approved gene therapies, the technique is still being investigated across a number of diseases to make sure that it will be safe and effective.
Our Approach to Developing Gene Therapies
With our platform technology, we create lysosomal enzymes that have increased ability to bind to the mannose 6-phopshate (M6P) receptor (the pathway to the lysosome), leading to improved biodistribution and efficient cross-correction of gene therapy products.
Specifically for gene therapies,
we incorporate two genes for the expression of both the lysosomal enzyme and the S1S3 phosphotransferase to ensure that the produced lysosomal will contain high M6P content. This results in produced therapeutic lysosomal enzymes with:
- Enhanced cellular uptake for cross-correction across a broad set of cells and tissues, and improved delivery of therapeutic enzyme to the lysosomes of otherwise enzyme-deficient cells
- More potent and effective treatments for LSDs
- Potential for using lower gene therapy dosages to mitigate safety and tolerability issues
This approach can potentially lead to more efficacious treatments with a lower therapy burden for diseases where there are already treatment options – meaning potentially just one dose of the therapy.