We are at the forefront
of a revolution in drug discovery targeting protein misfolding diseases
Natural forces chiseled every fold and fault over thousands of years, forming the landscape at Torrey Pines Beach. Similarly, protein folding and misfolding are strongly influenced by physical and cellular forces.
We use monogenic or pathway genetic evidence, bioinformatics, deep biology, and clinical path analysis to target unmet medical needs
High-Confidence Targets, Data Driven Analysis
Disease Modifying Focus, Innovative Discovery Platform
We are developing a pharmacological chaperone screening platform tailored for each specific disease target, anchoring on protein stability, coupled with phenotypic and functionality readouts that directly address disease defects
Specific and sensitive biomarkers will enable early patient idenfication, clinical stage readouts in target engagement and therapeutic efficacy
Biomarker Discovery, Precision Medicine
Protego Biopharma focuses on the discovery and development of first- or best-in-class small-molecule therapeutics that aim to reprogram protein folding for the treatment of various systemic amyloid diseases, such as light chain (LC) amyloidosis. We also target other monogenetic protein misfolding diseases that cause myopathy, cardiomyopathy, stroke, renal disease, retinal diseases, channelopathies, and various degenerative diseases. Our approach builds on the proven pharmacological chaperones approach previously exemplified by tafamidis, which was discovered and developed by our cofounders Dr. Jeffery W. Kelly and Dr. Richard Labaudinière, for the treatment of transthyretin amyloidosis.
We have assembled a proven team and joined forces with world-class investors and collaborators who embrace our mission.
Proteins are the workhorses of human biology. Integrity of protein structures is essential to protein’s physiobiological functions. Protein misfolding is increasingly recognized as an underlying cause of many chronic degenerative diseases, and may have two major consequences: 1) loss of biological function; 2) gain of toxicity or toxic function.
Protein homeostasis (proteostasis) is influenced by the energetics of protein folding, misfolding, and aggregation. Proteostasis is also affected by numerous regulated networks of interacting and competing biological pathways that control the biogenesis, folding, trafficking, and degradation of proteins present within and outside of the cells.
Protego’s technology tackles the protein misfolding problem through modulation of the energetics of protein or protein complexes by binding to small molecules such as pharmacological chaperones and/or by modulation of the cellular folding, secretory, and stress pathways. Protego’s approach builds on the proven pharmacological chaperones approach previously exemplified by tafamidis, discovered and developed by our cofounders Dr. Jeffery W. Kelly and Dr. Richard Labaudinière, for the treatment of transthyretin amyloidosis. The company also builds on the concept of correcting pathologic imbalances in protein homeostasis by specifically activating the unfolded protein response (UPR) with small molecules, to increase cellular folding capacity.
Protego Biopharma, Inc., a preclinical-stage biotechnology company dedicated to finding novel solutions for protein misfolding diseases, today announced it has raised $51 million in a Series A financing co-led by Lightspeed Venture Partners, Vida Ventures and MPM Capital.
Protego Biopharma receives a $250,000 award from Johnson & Johnson Innovation & JLABS. The funding will expedite the development of our specific and sensitive immunoassay for the detection of AL Amyloidosis.