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Developing Next Generation Therapies for Lysosomal Storage Disorders

A New Path

Unlocking the potential of , our breakthrough platform technology drives the development of next generation and for . Our mission is to translate the M6PT innovation into more effective next generation therapies for individuals affected by LSDs – to relieve suffering, to restore health, to increase hope, and to improve lives.

A New Path

Unlocking the potential of mannose 6-phosphate (M6P), our breakthrough platform technology drives the development of next generation enzyme replacement and for lysosomal storage disorders (LSDs). Our mission is to translate the M6PT innovation into more effective next generation therapies for individuals affected by LSDs – to relieve suffering, to restore health, to increase hope, and to improve lives.

Our Main Focus

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).

What Are Lysosomal Storage Disorders (LSDs)?

Lysosomal storage disorders (LSDs) are a group of more than 50 rare inherited diseases that are associated with life-threatening, life-shortening, and life-sapping complications. Most LSDs do not have treatments. While some do, the treatments may not adequately address the signs, symptoms, or progression of the disease for all individuals affected.

Mannose 6-Phosphate (M6P)

Mannose 6-phosphate (M6P) is a specialized carbohydrate structure found on lysosomal enzymes. M6P enables newly synthesized lysosomal enzymes to bind to M6P receptors in Golgi for delivery of the lysosomal enzymes to lysosome within cells. There are also M6P receptors on the cell surface of most, if not all cells, that can bind exogenous M6P-bearing lysosomal enzymes to enable their cellular uptake and delivery to lysosomes.

S1S3 Co-expression Platform Technology

Our innovative S1S3 co-expression platform technology enhances mannose 6-phosphate (M6P) content on lysosomal enzymes to increase lysosomal targeting to develop both enzyme replacement therapies (ERTs) and gene therapies.

Pipeline

We are developing next generation enzyme replacement therapies (ERTs) and gene therapies to treat LSDs and are working as quickly and efficiently as possible to meet the urgent needs of those living with these serious, life-threatening rare diseases.