WORLDSymposiumTM 2022 Satellite Symposium
February 2022
M6PT’s Chief Scientific Officer, Dr. Hung Do, discusses the importance of M6P and the potential to treat multiple lysosomal disorders using M6PT’s proprietary technology.
What Are Lysosomal Storage Disorders (LSDs)?
Lysosomal storage disorders (LSDs) are a family of 50 rare genetic, and life-threatening diseases characterized by a deficiency in a specific lysosomal enzyme. These disorders cause a buildup of substances such as lipids, carbohydrates, and proteins within cells’ lysosomal compartments, leading to problems that have significant systemic impact and can cause high morbidity and mortality.
Importance of Mannose 6-Phosphate (M6P)
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.
At M6P Therapeutics, we increase the M6P content, which enables delivery of enzymes to lysosomes more effectively.
Our Technology Platform
Our first-in-class bicistronic-S1S3 technology platform enhances M6P content on lysosomal enzymes for both recombinant enzyme and gene therapies, which improves enzyme uptake across target tissues, allowing us to restore the enzymatic activity within this natural pathway.
Our Development Pipeline
We have an extensive pipeline of programs to treat LSDs, beginning with Gaucher disease, Fabry disease, mucopolysaccharidosis IIIB (MPS IIIB) and mucolipidosis II (MLII).
Working at M6P
Join our team to work with high science and a breakthrough technology platform to develop therapies to improve the lives of patients with LSDs.