We see what’s possible and make it real.
We are developing a pipeline of first-in-class, next-generation enzyme replacement and gene therapies for lysosomal storage disorders (LSDs) by unlocking the potential of mannose 6-phosphate (M6P). We are working to develop life-changing therapies as quickly and efficiently as possible to meet the urgent needs of individuals living with these serious, rare LSDs.
Our initial research programs are focused on Gaucher disease, Pompe disease, Krabbe disease, mucopolysaccharidosis IIIB, Fabry disease, and alpha-mannosidosis. We expect to initiate two clinical programs by early 2022. Below please find a full pipeline of our development programs.