The Lost Enzyme Project Fund

$35,879.06 raised of $520,925.00 goal

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About

The Lost Enzyme Project was created by the families of children diagnosed with Beta-mannosidosis in the United States in collaboration with the University of California Irvine Foundation, Kimonis Laboratory, and JCR Pharmaceuticals to create a novel enzyme replacement therapy. Our project, working through the University of California Irvine Foundation, has set a goal of raising $520,925.00 by August 1, 2025, to fund the development of a new treatment with JCR Pharmaceuticals. Donate via Bright to ensure you get the Company match (!) but, if you would like more information, please visit the UCI Health's project website at https://fundraise.givesmart.com/e/o2kg0g?vid=114x2x.

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University of California Irvine Foundation

Research for The Lost Enzyme Project will take place at the Kimonis Lab at the University of California, Irvine. Our goal is to raise $520,925.00 by August 1, 2025, to fund the preclinical studies and research necessary to move into enzyme development with JCR Pharmaceuticals.

The project’s design is intended to support the following goals:

GOAL 1: Clinical characterization and natural history studies

GOAL 2: Utilization of Fribroblast cell lines for translational (treatment) studies

GOAL 3: Translational studies in neuronal cell lines derived from patient iPSCs (induced pluripotent stem cells)

GOAL 4: Conduct scientific lab work including a mouse model

We thank you for donating to our project. The groundbreaking enzyme replacement treatment will transform the outcomes of people diagnosed with Beta-mannosidosis around the world.

The Lost Enzyme Project Fund

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