Our Focus
Our Approach is Unique.
Our innovative S1S3 co-expression platform technology enables us to develop next generation, best-in-class, targeted enzyme replacement therapy (ERT) or gene therapy products to treat lysosomal storage disorders (LSDs)
Lysosomal enzymes created by our platform have increased levels of the specialized carbohydrate mannose 6-phosphate (M6P) to enable binding to the M6P receptor – the pathway to the lysosome – leading to improved biodistribution and lysosomal uptake of recombinant enzymes and efficient cross-correction of gene therapy products.
