A new collaboration between UCB and Refold Therapeutics aims to accelerate the development of antibody treatments for amyloidosis, a rare but often fatal condition where misfolded proteins build up in organs. The partnership highlights a growing trend of large pharmaceutical companies partnering with specialized biotech firms to translate complex scientific advancements into new therapies.
Understanding Amyloidosis
Amyloidosis encompasses more than 15 different forms, each caused by a different precursor protein that leads to amyloid formation and tissue deposition. As research in Nature Biotechnology details, these amyloid deposits can accumulate throughout the body, impacting organs like the heart, kidneys and nervous system. The specific form of amyloidosis targeted by this collaboration is light chain amyloidosis, the most common systemic form of the disease.
The Challenge of Light Chain Amyloidosis
In light chain amyloidosis, immune proteins misfold and clump together, depositing in vital organs. This buildup can lead to a range of debilitating symptoms, including breathlessness, swelling, and severe fatigue. Current treatment options often have limited success in removing the proteins once they’ve already accumulated, making early diagnosis and preventative measures crucial.
Refold’s Approach and the UCB Partnership
Refold Therapeutics, a spin-out from the University of Liverpool founded in , has secured an exclusive global license to develop antibodies from UCB that target misfolded “free light chains.” These rogue protein fragments are central to the pathology of light chain amyloidosis and also play a role in certain cancers, including hepatocellular carcinoma (liver cancer). The goal is to develop therapies that can both identify these harmful proteins earlier and neutralize them once they are present.
“Patients with light chain amyloidosis would benefit from an earlier diagnosis combined with improvements to their treatment,” said Tom Crabbe, CEO and Co-Founder of Refold Therapeutics. This sentiment underscores the significant unmet need for more effective treatments in this patient population.
Beyond Amyloidosis: Potential in Oncology
The collaboration isn’t limited to amyloidosis. Refold plans to investigate the role of free light chains in driving tumor growth in cancers like liver cancer. If successful, this could open up a new avenue for treatment in oncology, leveraging the same antibody constructs developed for amyloidosis.
The Deal Details
The agreement between UCB and Refold involves an upfront payment from UCB, along with potential milestone payments and royalties on future sales. Refold will take the lead in transforming the antibody constructs into clinical-stage drugs. Alistair Henry, Executive Vice President and Chief Scientific Officer at UCB, emphasized the value of such licensing agreements, stating they demonstrate the strength of UCB’s scientific innovation and research productivity.
A Growing Trend in Pharmaceutical Collaboration
This partnership exemplifies a broader industry trend of large pharmaceutical companies out-licensing early-stage assets to nimble biotech firms. This allows larger companies to maintain a broad pipeline without bearing the full cost and risk of developing every project in-house. It also allows specialized biotechs, like Refold, to focus their expertise on specific disease areas and accelerate the development of innovative therapies.
Liverpool’s Rising Biotech Hub
The success of Refold is also a positive development for the Liverpool region, signaling a shift in the UK life sciences landscape. Backed by LYVA Labs, Liverpool’s regional innovation vehicle, Refold joins a growing number of spin-outs translating university research into commercial ventures. This demonstrates that significant drug discovery and deal-making are now occurring beyond the traditional “Golden Triangle” of London, Oxford, and Cambridge.
What This Means for Patients
The collaboration between UCB and Refold offers a glimmer of hope for patients with light chain amyloidosis and potentially other cancers. By focusing on early detection and targeted neutralization of misfolded proteins, this research aims to improve survival outcomes and quality of life for those affected by these challenging conditions. While the development of new therapies is a lengthy process, this partnership represents a significant step forward in addressing the unmet needs of patients with amyloidosis and exploring new treatment avenues in oncology.
