Our Platform Technology
We Improve on Nature.
Our innovative S1S3 co-expression platform technology enhances mannose 6-phosphate (M6P) content on lysosomal enzymes to increase lysosomal targeting to develop both enzyme replacement therapies (ERTs) and gene therapies. Simply put, these molecules can bind with greater affinity to the specific receptor, thereby improving enzyme uptake and lysosomal delivery across target tissues, which can restore the enzymatic activity within this natural pathway.
With our S1S3 platform technology, we have the capability to develop next generation, best-in-class, targeted ERT and gene therapy products, improving their ability to get to affected cells and organs.
Since the platform regulates the M6P levels on lysosomal enzymes, we can potentially treat even those LSDs with enzymes that have naturally low M6P levels, such as mucopolysaccharidosis IIIB (MPS IIIB) and alpha-mannosidosis.
M6PT’s Platform Technology Overview
The below animation provides an overview of M6P Therapeutics’ bicistronic-S1S3 technology platform, how it works in target lysosomal storage disorders, and its potential.
Our S1S3 platform technology enables us to:
- Deliver therapies more efficiently – and with more precise targeting to lysosomes – which means potentially less medication is needed than other treatments.
- Enhance in vivo phosphorylation cross-correction, so LSD enzymes can be taken up and targeted to the lysosomes of otherwise enzyme-deficient cells.
- Develop therapies that reach more affected organs and cells, which may lead to new therapies for currently untreated diseases.
- Produce a next generation approach with advantages over first-generation ERT and gene therapy products.
- Take advantage of a strong intellectual property (IP) position with global rights and the freedom to operate.