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Leadership

We Have Proven Expertise in Rare Disease Drug Development.

Our leadership team has collectively more than 100 years of experience and deep expertise in the discovery, non-clinical, clinical, and commercial development of drugs that treat rare diseases, particularly lysosomal storage disorders (LSDs)

Our team appreciates the unmet medical need for those affected by LSDs and is committed to the promise of enzyme replacement therapies (ERTs) and gene therapies by harnessing the power of mannose 6-phosphate (M6P) with our innovative S1S3 co-expression platform technology.

Drawing from relevant science, non-clinical and clinical development, regulatory affairs, manufacturing, commercial operations, financial and general management, and other vital functions, we apply our experience to develop next generation therapies, whether enzyme replacement or gene therapies, for LSDs. We make, implement, and critique decisions with efficiency and accountability, drive to our stated objectives, and make course corrections as warranted.

Cuong Do

Founder and Chairman

Stuart Kornfeld

Washington University. Cofounder, M6PT & Chair, SAB

Cuong Do

Founder and Chairman

Stuart Kornfeld

Washington University. Cofounder, M6PT & Chair, SAB