Overview

Cantabio’s ability to generate a pipeline of high quality therapeutic candidates stems from its integration of key elements and novel discovery techniques, combined with years of experience in the field. Cantabio strategically integrates therapeutic focus, target family biophysics, novel drug discovery technologies and expertise into an innovative drug discovery approach, producing a constant flow of therapeutic candidates with real-world applications for a number of related diseases and ready for clinical development.

Identifying and Developing Pharmacological Chaperones as Disease Modifying Therapeutic Candidates

Our small molecule pharmacological chaperones act to stabilize the native functional form of selected protein targets against misfolding when they lose their function and/or become toxic and thus trigger cell death. Our pharmacological chaperones are identified using a unique biophysics based screening platform. Upfront pharmacological characterization of discovered compounds in cellular models of the targeted disease ensures that only those that are therapeutically functional with ideal CNS properties are taken forward and developed into leads, using a synergistic process of structural biology, computational chemistry and medicinal chemistry. The leads are further optimized through validated neurodegenerative disease related cellular and in vivo models to develop disease-modifying drug candidates for clinical trials.


Pharmacological chaperone binds folded structure of a protein to stabilize its native functional form and reduce its destabilization, misfolding and aggregation.

Engineered CNS-penetrant Protein Technology

As a complementary approach, we are pursuing the application of protein delivery technologies, which enables the delivery of targeted proteins into patients' brain to supplement their lowered functional in vivo levels.

Covalent attachment of cell permeable peptides to proteins enables their delivery into patients’ cells and central nervous system to supplement low levels of the protein. At Cantabio we are applying this protein delivery technology by engineering CNS-penetrant protein therapeutic candidates.

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Therapeutic Targets

Our first therapeutic target is the DJ-1 protein. Loss of and reduced function of the DJ-1 protein has been linked to the onset of a variety of diseases such as Parkinson’s disease, Alzheimer’s disease and more. The therapeutic targeting of DJ-1 could yield added mechanistic benefits, such as protection from oxidative stress and protein misfolding and aggregation.

Our second therapeutic target is the Aβ peptide. The aggregation and amyloid formation of the Aβ peptide is linked to the onset and progression of Alzheimers disease. Cantabio’s small molecules bind to monomeric Aβ peptide and aim to reduce the misfolding and aggregation of Aβ peptide through stabilization and promotion of its functional native states.

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