Many diseases are caused by the excess accumulation of a toxic protein or metabolite
Diabetic retinopathy Rheumatoid arthritis Diabetic complications Coronary artery disease
Lysosomal storage disease
Aging
Uremia
Kidney failure Phenylketonuria
ATTR amyloidosis
We focus on diseases with unmet clinical need, a clear mechanism of action (MOA), and for which an enzyme therapy is the most efficacious therapeutic modality.
