Protego Raises $130 Million to Fund AL Amyloidosis Pivotal Study

Rare Daily Staff

Protego Biopharma, which is pioneering first-in-class small-molecule therapeutics that reprogram protein folding to address systemic amyloid diseases and other protein misfolding disorders, announced it has completed an oversubscribed $130 million series B financing.

Novartis Venture Fund and Forbion led the round, which included participation from new investors Omega Funds, Droia Ventures, YK Bioventures, and Digitalis Ventures. Existing investors, including Vida Ventures, MPM BioImpact, Lightspeed Venture Partners, and Scripps Research, also participated.

The proceeds will be used to advance Protego’s lead experimental therapy, PROT-001, into a pivotal clinical trial for AL amyloidosis, a rare and often fatal condition caused by protein misfolding that leads to organ damage, especially in the heart.

Protego’s therapeutic strategy is rooted in human genetics and a unique pharmacological chaperone mechanism. These small molecules act as cellular guides, ensuring proteins fold correctly. By stabilizing immunoglobulin light chains and preventing amyloid buildup, PROT-001 targets the underlying cause of disease, rather than simply managing symptoms.

The approach represents a potential paradigm shift for AL amyloidosis and a wide spectrum of protein misfolding disorders with significant unmet needs.

“Protego is advancing a highly differentiated approach with the potential to transform treatment for patients with AL amyloidosis,” said Tim Lohoff, principal at Forbion. “By targeting the root cause of this severe disease, PROT-001 has the potential to deliver the first truly disease-modifying therapy in this indication”