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Leveraging our unique expertise in neurodegenerative diseases to develop better therapeutics

Pipetting Samples

Our first strategy to fix Traffic Jams in the Brain

Manipulating O-GlcNAc signaling via O-GlcNAc inhibition is a promising route for therapeutics to address progressive neurodegenerative diseases that is supported by scientific data. 

O-GlcNAcylation is essential to preserve nuclear pore integrity, therefore increasing levels of

O-GlcNAc has the potential to prove therapeutic for neurodegenerative diseases that have shown decreased levels of O-GlcNAc in the nuclear pore complex and nucleocytoplasmic transport.

Prior studies have demonstrated that increasing levels of O-GlcNAc through inhibition of OGA utilizing the inhibitor Thiamet-G mitigates levels of neurotoxicity in numerous models of Alzheimer's disease. 

Furthermore, applying OGA inhibition via Thiamet-G has been shown to correct nucleocytoplasmic transport defects and neurotoxicity in models of Huntington's disease. 

Mounting evidence suggests that increasing levels of O-GlcNAc through inhibition of OGA may provide an avenue for correcting nucleocytoplasmic transport defects and mitigating neurotoxicity in neurodegeneration - which merits further scientific investigation.

Our OGA Inhibitors

We have two structurally unique OGA inhibitors that are currently undergoing pre-clinical optimization and pre-clinical efficacy and safety studies in parallel. 

The unique characteristics of our small molecule inhibitors have a PK profile that supports once a day dosing, exhibit strong brain penetrant attributes and target inhibition at nanomolar concentrations. 

The optimal brain-to-plasma concentration ratio enables maximum target engagement with minimal off-target toxicities.

The IND enabling toxicology is projected to start early 2023, with IND filing projected in Q4 of 2023. Our lead target indications for our lead compound are ALS and FTD.

Our OGA inhibitors belong in the 3rd generation class, as they both have a novel molecule structure that is designed with intimate knowledge of OGA and SAR within this unique class of OGA inhibitors. 

Both compounds are more potent than existing OGA inhibitors, providing better binding specificity towards OGA and likely translating into less off-target binding and an enviable safety profile. 

Furthermore, there has been proof-of-concept preclinical data in human induced pluripotent stem cells, post-mortem human brain tissue, drosophila, mouse models and other methods to support the lead indication selection of ALS and FTD.

Our Pipeline

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Image by Josh Appel

Neurodegenerative Diseases

Understanding complex and devastating neurodegenerative diseases

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Traffic Jams in  Neurodegeneration

How exciting is the linkage between both the NPC and NCT with neurodegeneration?

Pipetting Samples

Informed Pipeline

Making data informed decisions to build a robust pipeline

DNA

TM

PurPrecision    Drug Discovery Platform

Building a platform that uses lessons from our novel discovery for the future