A New Era in Healthcare Innovation
At the heart of one of Europe’s most dynamic forums for drug repurposing, the International Drug Repurposing Conference 2025 (iDR25), the DREAMS project showcased how artificial intelligence (AI) is redefining therapeutic development for rare diseases. Held at the iconic Felix Meritis in Amsterdam and hosted by REMEDi4ALL and Beacon for Rare Diseases, iDR25 brought together leading minds to explore scalable solutions for some of medicine’s most urgent challenges.
DREAMS (Drug Repurposing with Artificial Intelligence for Muscular Disorders), an ambitious Horizon Europe project, was strongly represented at the event, reinforcing its position as a driver of cutting-edge innovation at the crossroads of biotechnology, data science, and patient-centered research.
Why DREAMS Matters: Tackling the Untreated
With over 600 identified neuromuscular disorders (NMDs) and only 11 approved treatments, the landscape of rare disease therapy is marked by a critical gap in innovation. DREAMS addresses this unmet need by developing a reusable platform to discover therapies that can simultaneously target multiple NMDs — and potentially, other rare diseases sharing common biological signatures.
Using a combination of induced pluripotent stem cell (iPSC) technology and AI-driven drug discovery, DREAMS is charting a faster, smarter route to drug development. The project focuses on five initial NMDs sharing defects in autophagy and desmin disorganization, with the goal of identifying shared biomarkers and drug targets that unlock broader therapeutic potential.
Highlights from iDR25: Thought Leadership and Knowledge Exchange
A Strong Presence from the DREAMS Consortium
DREAMS was represented at iDR25 by leading members of its consortium:
- Xavier Nissan (I-Stem)
- Wael Khazen & Alexandre Mejat (AFM-Téléthon)
- Nicolas Maignan (Kantify)
Their participation underscored the consortium’s deep expertise in fields ranging from stem cell biology to machine learning and translational research.
Pushing the Frontiers of AI for Drug Repurposing
One of the standout moments was the presentation by Nicolas Maignan (Kantify) during the “AI in Drug Repurposing” session. His talk explored how the DREAMS project leverages multi-layered AI models to accelerate discovery and validation of new therapies:
- A first AI model processes large-scale screening data from iPSC-derived skeletal muscle cells to identify shared drug targets.
- A second model identifies promising drug candidates, including repurposable drugs and new molecular entities (NMEs).
- A third model expands the scope by identifying additional diseases that share drug targets with the initial five NMDs.
This end-to-end AI pipeline is not only accelerating drug discovery but also enabling multi-indication development strategies, a game-changer in the field of rare diseases.
Building Bridges Across Sectors
iDR25 served as a timely reminder that advancing treatments for rare diseases cannot happen in silos. DREAMS’ approach — combining the strengths of academic research institutions, biotech innovators, AI specialists, and patient organizations — exemplifies the collaborative model needed to overcome systemic barriers in orphan drug development.
With 9 partners across 6 countries, DREAMS brings together deep domain knowledge with technological prowess, making it uniquely positioned to deliver impact both within and beyond the project’s initial scope.
Why Events Like iDR25 Are Crucial
Beyond the technical discourse, events like iDR25 play a strategic role in creating a shared vision for the future of healthcare innovation. For projects like DREAMS, they are vital platforms to:
- Engage with regulators, policymakers, and payers on sustainable models for rare disease treatment.
- Align with industry and academic partners on data interoperability, ethical standards, and AI validation.
- Ensure that patient perspectives remain central in the innovation pipeline.
DREAMS’ presence at iDR25 reaffirmed its commitment to these goals and highlighted its potential to contribute to a pan-European transformation in drug development for rare diseases.
Looking Ahead: Toward Scalable Impact
With a total budget of €7.8 million and a timeline extending to 2028, DREAMS is more than a research project — it is an innovation ecosystem. The insights shared at iDR25 reflect not just current progress but the promise of a paradigm shift in how we approach complex, underserved medical conditions.
As the consortium moves toward the discovery, preclinical validation, and clinical trial preparation of its lead candidates, the ripple effects of DREAMS are already shaping a more inclusive and efficient biomedical future.
