Protego Biopharma is a San Diego-based, clinical-stage biotechnology company developing first-in-class small molecule pharmacological chaperones to treat systemic amyloid diseases and other protein misfolding disorders.
Protego Biopharma is a clinical-stage biotechnology company that develops first-in-class small molecule therapeutics designed to treat protein misfolding diseases. The company uses an integrated approach combining protein sciences, advanced computation, and medicinal chemistry to discover pharmacological chaperones. These molecules function as cellular guides that stabilize proteins, such as immunoglobulin light chains, preventing them from misfolding and aggregating into toxic deposits. This mechanism aims to address the root causes of systemic amyloid diseases rather than managing symptoms, building upon the foundational research of co-founders Dr. Jeffery W. Kelly and Dr. Richard Labaudinière.
The company's lead program, PROT-001, targets AL amyloidosis, a rare and potentially fatal condition where misfolded light chain proteins accumulate in vital organs. Protego is advancing this candidate into pivotal clinical trials following Phase 1 studies. By integrating precision medicine and proprietary biomarker discovery tools like AmyLite, the company seeks to improve patient stratification and therapeutic efficacy. Headquartered in San Diego, Protego is backed by a group of life sciences investors and is focused on delivering disease-modifying therapies for patients suffering from critical unmet medical needs.