Our mission is to meet the large unmet need and accelerate the availability of treatments for patients and families living with lysosomal storage disorders.
With a patient-first approach in our R&D process, Polaryx is committed to developing non-invasive therapeutic alternatives to treat lysosomal storage disorders. Our orally-available small molecule therapies aim to promote lysosome biogenesis via PPARα-dependent TFEB upregulation, attenuate inflammation in the brain, and enhance neuronal survival. Our gene therapy candidate is formulated as an intranasal administration and planned for administration in combination with our small molecule therapies.
In collaboration with Rush University Medical Center, we have confirmed the in-vivo efficacy of our drug candidates using human astrocytes and various murine models of lysosomal storage disorders. Importantly, all of our preclinical studies improved motor and cognitive functions and increased life expectancy.
