In December 2025, the European Rare Diseases Research Alliance (ERDERA) announced that a collaborative transnational PSC research project was funded. This project, named ASCENT-PSC, is led by a Canadian Toronto-based team.
ERDERA’s investment in rare disease research includes an expectation of genuine patient-partnership. The research team is led by Sonya MacParland at University Health Network and includes Principal Investigators from Canada, Germany, France, Netherlands, Norway, and Turkey (Espen Melum, Richard Post ,Thomas Baumert, Adrien Guillot, and Esra Erda). PSC PARTNERS SEEKING A CURE CANADA and Mary Vyas will be serving as the Patient Advocacy Organization (PAO) research partner with collaboration from Stephen Rossi at PSC PARTNERS SEEKING A CURE.
ASCENT‑PSC builds an atlas and pre‑clinical model platform for primary sclerosing cholangitis, a rare liver disease currently without approved medical therapies other than transplantation. By integrating genomic, transcriptomic and proteomic data with precision‑cut liver slices, human liver organoids and microbiome‑modified mouse models, the consortium will identify and functionally test promising therapeutic targets.
From ERDERA’s LinkedIn Announcement
The results of the Joint Transnational Call 2025 are out 🎉 ERDERA has announced the 18 selected projects under its first Joint Transnational Call on preclinical therapy studies for rare diseases using small molecules and biologicals 🧬
From 161 submitted proposals, 18 transnational projects were selected for funding, representing a combined investment of approximately €29 million 🔬. These consortia bring together partners supported by 29 national and regional funding organisations across 23 countries, co-funded by the European Commission 🇪🇺🤝
Each project will contribute robust pre-clinical evidence to support the transition towards future clinical trials, with a shared objective: safer and more effective therapies for people living with a rare disease 💙
👉 Discover all selected projects and how they address the call’s core aims at https://loom.ly/BAShwKw.
Targeting PSC using Human Pre-clinical Models and Spatial Transcriptomics
From ERDERA’s website Joint Transnational Call 2025 page:
Partners
- MacParland, University Health Network (Canada)
- Erdal, Izmir Biomedicine and Genome Center (IBG) (Türkiye)
- Melum, University of Oslo Faculty of Medicine (Norway)
- Baumert, Institute of Translational Medicine and Liver Disease, Inserm U1110 (France)
- Guillot, Charité – Universitätsmedizin Berlin (Germany)
- Post, Erasmus MC (Netherlands (the))
- Vyas, PSC Partners Seeking a Cure Canada (Canada)
Scientific abstract
Challenge: Our translational and patient-partnered team studies primary sclerosing cholangitis (PSC), a rare liver disease with no approved treatments and a significant unmet medical need. Due to the limited patient population and no speedy pathway for regulatory approval, the case for for-profit investment in drug development for PSC is challenging. This proposal aims to develop new treatments for this rare disease.
Solution: To address this gap, we have developed the first transcriptomic map of the PSC liver, providing an unprecedented view of disease mechanisms at the cellular level. We have analyzed these disease mechanisms and pathways to identify known targets and their corresponding drugs. This analysis, along with expert review, identified immune modulation as an avenue to target immune-mediated fibrotic pathways within the PSC liver.
There is no completely reliable animal model of PSC for pre-clinical testing, so we will optimize mouse models and then examine the similarities between animal and human disease. We will also employ ex vivo 3D human tissue platforms using organoids and precision cut liver slices to evaluate the efficacy of small molecule interventions in human PSC liver tissue.
We will then test the ability of immune therapies to modulate fibrogenic processes in the optimized mouse models and human explanted PSC tissue in comparison to healthy human liver using spatial transcriptomics and proteomic profiling as a readout to test for normal liver function after disease condition treatment.
Expected Achievements/Impact: This work will be transformative and we will collectively make new discoveries that will translate to new options for rare liver disease patients. The patient-partnered framework is a novel approach to research that puts patients in the driver seat of the drive to new treatments for their disease.
This work will also transform the rare disease research landscape from redundant research silos to a collaborative consortium.
