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Protego Biopharma develops first-in-class small molecule therapeutics by reprogramming protein folding for various protein misfolding diseases. It uses pharmacological chaperones to modulate target protein stability, restoring function and re-establishing proteostasis via cellular stress pathway modulation. This targets conditions like myopathy, cardiomyopathy, renal disease, and other degenerative disorders.
Founded by Dr. Jeffery W. Kelly and Dr. Richard Labaudinière, Protego Biopharma builds on prior success with tafamidis, a pharmacological chaperone for transthyretin amyloidosis. This demonstrated protein folding modulation efficacy. Protego’s strategy now activates the unfolded protein response with small molecules, correcting protein homeostasis imbalances.
Protego Biopharma targets patients with chronic degenerative diseases from misfolded proteins, addressing unmet medical needs. Its vision is to accelerate novel treatment development to restore cellular proteostasis. Protego aims to provide transformative solutions, improving outcomes by managing underlying pathologies of these challenging illnesses.
Protego Biopharma has raised $181.0M across 2 funding rounds.
Protego Biopharma has raised $181.0M in total across 2 funding rounds.
Protego Biopharma has raised $181.0M across 2 funding rounds. Most recently, it raised $130.0M Series B in December 2025.
| Date | Round | Lead Investors | Other Investors | Status |
|---|---|---|---|---|
| Dec 1, 2025 | $130M Series B | TIM Lohoff, Novartis | Digitalis Ventures, Droia Ventures, Lightspeed Venture Partners, MPM Capital, Omega Funds, Scripps Research, Christopher Weyrer, MD, PHD, YK Bioventures | Announced |
| Oct 1, 2021 | $51M Series A | MPM Capital, Vida Ventures, Shelley CHU | Atlas Venture, Nextech Invest | Announced |
Protego Biopharma has raised $181.0M in total across 2 funding rounds.
Protego Biopharma's investors include Tim Lohoff, Novartis, Digitalis Ventures, Droia Ventures, Lightspeed Venture Partners, MPM Capital, Omega Funds, Scripps Research, Christopher Weyrer, MD, PhD, YK Bioventures, Vida Ventures, Shelley Chu.
Protego Biopharma is a clinical-stage biotech company headquartered in San Diego, California, specializing in the discovery and development of first-in-class small-molecule therapeutics that reprogram protein folding to treat protein misfolding diseases.[1][2][3][4] It targets conditions like AL amyloidosis, myopathy, cardiomyopathy, stroke, renal disease, retinal diseases, channelopathies, and degenerative diseases using pharmacological chaperones—small molecules that stabilize misfolded proteins, building on the success of tafamidis for transthyretin amyloidosis.[1][4][5] The company serves patients with rare and systemic diseases lacking effective treatments, solving the core problem of protein misfolding by restoring protein homeostasis and enabling precision medicine through biomarkers for early detection and efficacy readouts.[4][5] Recent growth includes a $130 million oversubscribed Series B financing in 2025 to advance its lead candidate, PROT-001, into pivotal trials for AL amyloidosis, marking strong momentum toward disease-modifying therapies.[5]
Protego Biopharma emerged from the expertise of its cofounders, Dr. Jeffery W. Kelly and Dr. Richard Labaudinière, who previously discovered and developed tafamidis—the first marketed disease-modifying agent for transthyretin amyloidosis using the pharmacological chaperone approach.[1][4] The company, based in San Diego with fewer than 25 employees and revenue under $5 million, was formed to expand this proven strategy to a broader range of protein misfolding diseases, incorporating advances in biology, chemical biology, biomarkers, structural biology, and computation.[1][4] Early traction stems from human genetics, bioinformatics, and deep biology to select high-confidence targets, culminating in the clinical-stage PROT-001 program and the recent $130 million Series B led by investors like Forbion, validating its mission with world-class collaborators.[4][5]
Protego rides the wave of precision medicine and protein homeostasis therapeutics, targeting protein misfolding—a key driver of rare diseases and neurodegeneration amid rising demand for disease-modifying drugs beyond symptomatic relief.[2][4][5] Timing is ideal post-tafamidis approval, with genetic insights and AI-driven biology accelerating target validation in a market hungry for AL amyloidosis solutions, where PROT-001 could shift paradigms by halting amyloid progression.[5] Favorable forces include biotech funding resurgence for genetics-backed platforms and unmet needs in monogenic diseases, positioning Protego to influence the ecosystem by validating chaperones/UPR modulation for broader applications like retinal and renal disorders.[4][5]
Protego is primed to deliver PROT-001 as the first disease-modifying therapy for AL amyloidosis via pivotal trials, funded by its $130 million Series B, with potential expansion to pipeline targets in myopathy, cardiomyopathy, and beyond.[5] Trends like biomarker-enabled precision oncology and UPR-targeted proteostasis will propel its platform, evolving its influence from niche rare diseases to systemic disorders as clinical data de-risks follow-on assets. This builds directly on its high-level mission, offering hope where few options exist today.[4][5]
