We harness the potential of mannose 6-phosphate (M6P).
Mannose 6-phosphate (M6P) is a specialized carbohydrate structure found on the surfaces of lysosomal enzymes, and it enables enzyme replacement therapy (ERT) enzymes to bind to specialized M6P receptors on cell surfaces. Once this happens, the ERT enzymes can be delivered to the lysosomes to exert their biological function of metabolizing the accumulating substrates. M6P is required to deliver ERT enzymes to lysosomes. With low levels of M6P, the enzymes are not able to reach the lysosomes as effectively.
Historically, it has been extremely difficult to create recombinant enzyme therapies with high amounts of M6P. And, from a gene therapy perspective, it is virtually impossible to control carbohydrate processing in vivo. Prior efforts to enhance M6P content have largely failed. Consequently, most ERTs and gene therapy products do not reach the lysosomes where they are needed in sufficient quantities.
Our team of scientists are experts in rare disease drug development, particularly lysosomal storage disorders (LSDs) and M6P research, have developed a technology platform to increase the M6P content of ERT enzymes to improve lysosomal targeting and delivery.
Because these M6P has greater affinity to bind to the M6P receptors on the lysosome, we are able to develop targeted, individualized, and effective medicines, either ERT or gene therapy, to treat the specific LSD. We also have the opportunity to improve treatment options for other LSDs that currently have approved therapies.
Our initial research programs are focused on Gaucher disease, Fabry disease, Pompe disease, and mucolipidosis II (MLII). Learn more about our pipeline of LSD programs. Learn more about our pipeline of LSD programs. We plan to enter the clinic with our first clinical program in 2022.