About the Research

Unlocking Hope: Join the Challenge to Fund Gene Therapy for Mucolipidosis II/III A Bold Investment. A Rare Opportunity. A Life-Saving Impact.

We have launched a Challenge Grant campaign to fund a historic breakthrough: the first-ever gene therapy for Mucolipidosis II/III (ML II/III), an ultra-rare and

fatal disorder with no approved treatment. Our goal is to raise $500,000 by December 2027, with critical early funding milestones needed in the next 12-18 months to ensure research efforts can proceed on schedule.

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At the heart of this campaign is our commitment to fully fund a $500,000 research grant for Dr. Patricia Dickson, a world-renowned leader in lysosomal storage disorder (LSD) research. Dr. Dickson has over 20 years of experience working with the LSD community, and her lab is among those leading the charge in pioneering gene therapies for clinical development. Her project, “Gene Therapy for Mucolipidosis II/III with AAV S1S3 Phosphotransferase,” offers a first real path toward treatment for the ML II/III community, using a proven viral vector to deliver a corrected gene directly into cells. Why This Matters ML II/III robs children of speech, mobility, and cognition. Most with ML II/III do not survive childhood, and families are often left behind by traditional research funding due to the disease’s rarity. This Challenge Grant is a source of hope for ML families, and an opportunity to unite community power with scientific promise.

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The gene therapy will utilize novel properties that make this program truly unique. The use of a hyperactive phosphatase (S1S3) which replaces the missing protein in ML kids is 20x more active in cells. S1S3 is an engineered enzyme that Dr. Stuart Kornfeld from WashU developed several years ago that is much more active and much smaller which allows it to be efficiently packaged inside a gene therapy vector.