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.
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.