Our Approach
There is increasing recognition that therapeutic targets representing central hubs of disease pathogenesis are required to slow disease progression in a clinically meaningful way. As a central hub of disease, the endolysosomal system represents a means of targeting multiple disease mechanisms at once.
We aim to enhance endolysosomal transport by improving the function of the retromer pathway. This approach can potentially protect against or reverse neurodegenerative diseases such as Alzheimer’s Disease, Parkinson’s Disease, and ALS.
Complementary strategies to restore endolysosomal function
1
Stabilizing the retromer complex
We are developing compounds to stabilize retromer subunits and prevent the disassembly of the complex.
1
2
Increasing levels of retromer core subunits
We are developing therapies that can restore the amount of functional retromer complex in the cell. By raising retromer protein levels, we can increase or replace defective retromer protein subunits.
2
3
Enhancing SORL1 function
As the link between the retromer trimeric core and its cargo, SORL1 is necessary for cargo transportation. We are working on therapies that will enhance SORL1 function for more efficient protein sorting and transport.
3
Stabilizing the retromer complex
We are developing compounds to stabilize retromer subunits and prevent the disassembly of the complex.
