What Are Lysosomal Storage Disorders (LSDs)?

Lysosomal storage disorders (LSDs) are a family of 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 affect multiple organs.

Importance of Mannose 6-Phosphate (M6P)

M6P enables delivery of enzymes to lysosomes, which is an important part of being able to develop therapies for patients with these serious LSDs.

 

Our Technology Platform

Our first-in-class technology platform creates lysosomal enzymes that harness high levels of M6P, allowing us to restore the enzymatic activity within the natural pathway for exogenous enzymes (enzyme replacement therapy or ERT) and gene delivery.

 

Our Development Pipeline

We have an extensive pipeline of programs to treat LSDs, beginning with Gaucher disease, Pompe disease, Krabbe disease, mucopolysaccharidosis IIIB, Fabry disease, and alpha-mannosidosis.

Working at M6P

Join our team to work with high science and a breakthrough technology platform to develop therapies to improve the lives of all patients with LSDs.