The Lost Enzyme Project is a Non-profit campaign created by the families of those with Beta-mannosidosis. Beta-mannosidosis is a progressive lysosomal disorder with no treatment. Patients and their families have struggled to find hope for this under-researched and poorly understood disease. The Lost Enzyme Project was created by the families of those patients diagnosed with Beta-mannosidosis the United States, in collaboration with Kimonis Lab at the University of California, Irvine to complete the preclinical studies necessary for the development of an enzyme replacement therapy for Beta-mannosidosis. 

JCR Pharmaceuticals has partnered with Kimonis Lab to develop beta-mannosidase, the enzyme lost in beta-mannosidosis patients, utilizing its patented, state-of-the-art J-Brain Cargo® technology. J-Brain Cargo® is the first technology proven to penetrate the blood-brain barrier to deliver treatment to the central nervous system in humans. The experimental therapeutic will be used to test its utility in an animal model of beta-mannosidosis as well as human cells with the goal of creating human clinical trials.

To date, we have raised just over $160,000.00 through the UCI Foundation towards this goal. These funds have facilitated on-site training with JCR in Japan, as well as the preliminary work in cell line and animal studies. 

We are a grassroots campaign, funded by kind hearts like yours.