What We Do
We aim at accelerating the availability of treatment options for underserved patients.
Lysosomes are cellular membrane-bound acidic organelles that perform a pivotal function in cellular homeostasis by regulating degradative pathways with hydrolytic enzymes. Aberrant lysosomal function leads to the pathogenesis of lysosomal storage disorders. Thus, augmentation of the cellular clearance pathways by induction of lysosome biogenesis is an attractive therapeutic intervention for both lysosomal storage disorders and neurodegenerative disorders in which toxic aggregates are responsible for the disease pathogenesis.
Based on this rationale, we are developing patient-friendly, orally-available small molecule therapeutics. Our core platform aims to boost lysosome biogenesis via PPARα-dependent TFEB upregulation.This approach will contribute to developing the right solutions to help patients suffering from lysosomal storage disorders and neurodegenerative disorders.
In collaboration with Rush University Medical Center, we have fully confirmed the in vivo efficacy of our drug candidates using various murine models of lysosomal storage disorders, including Late Infantile Neuronal Ceroid Lipofuscinosis, Juvenile Neuronal Ceroid Lipofuscinosis, Krabbe disease, Sandhoff disease, and Niemann Pick Type A/B disease, as well as neurodegenerative disorders, such as Alzheimer’s disease and Parkinson’s disease via oral administration of our candidate drugs, PLX-100, -200, and -300. Importantly, all of these disease murine models showed improved motor and cognitive functions for Late Infantile Neuronal Ceroid Lipofuscinosis, Juvenile Neuronal Ceroid Lipofuscinosis, Alzheimer’s disease, and Parkinson’s disease.
Our patient-friendly, oral, small molecule therapeutics may provide patients with a noninvasive treatment option that can be taken either alone or in combination with other possible treatments. Furthermore, because one of our drug candidates uses a mixture of repurposed drugs, we will be able to bring safe therapeutic interventions to patients quickly.
Drug repurposing saves time, cost,
and generally provides extensive
information on safety.