Lysosomal storage diseases (LSDs) comprise a class of almost fifty inherited metabolic disorders that may affect different parts of the body, including the skeleton, brain, skin, heart, and central nervous system. They are caused by an abnormal build-up of toxic metabolites in lysosomes as a result of enzyme deficiencies.
Takeda is a world leader in developing therapies to treat LSDs. We are currently seeking small molecule binders of glycosaminoglycans and/or lysosome associated proteins to further our therapeutic studies. We will use these small molecules to develop PET (positron emission tomography) ligands to assess the efficacy of various treatments for multiple LSDs, first in animal studies and then in humans. We would also welcome computational screening platforms that could identify such small molecules.
These PET ligands will be instrumental for our studies to understand how our therapies are affecting the pathology of the disease in real time, particularly in children and in the brain.
Deadline: July 31, 2022
Funding ranges from $150,000 - $300,000 over 1-2 years for further evaluation of potential leads along with possible license of the researcher’s patented materials.